FDA Updates

By Coping Magazine 26 Mar, 2024 CANCER, UNDERSTANDING CANCER CANCER TREATMENT, COPING WITH CANCER, FDA

Recent Oncology/Hematology Approvals from the U.S. Food and Drug Administration

FDA Approves Elahere for FRα Positive, Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
The Food and Drug Administration approved ImmunoGen’s Elahere (mirvetuximab soravtansine-gynx) for adults with FRα positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. People are selected based on an FDA-approved test. Elahere previously received accelerated approval for this indication. This application was granted priority review. (March 2024)

Iclusig Gets FDA Accelerated Approval with Chemotherapy for Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
Takeda’s Iclusig (ponatinib) was granted accelerated approval with chemotherapy for adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

This application was granted priority review and orphan drug designation. (March 2024)

FDA Grants Accelerated Approval to Brukinsa for Relapsed or Refractory Follicular Lymphoma
The FDA granted accelerated approval to BeiGene for Brukinsa (zanubrutinib) with obinutuzumab for relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

This application was granted fast track designation and orphan drug designation. (March 2024)

Opdivo Receives FDA Approval in Combination with Cisplatin and Gemcitabine for Unresectable or Metastatic Urothelial Carcinoma
The FDA approved Bristol-Myers Squibb’s Opdivo (nivolumab) in combination with cisplatin and gemcitabine for first-line treatment of adults with unresectable or metastatic urothelial carcinoma (UC).

For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (March 2024)

FDA Approves Besponsa for Children with Acute Lymphoblastic Leukemia
Pfizer’s Besponsa (inotuzumab ozogamicin) for children 1 year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). This application was granted priority review and orphan drug designation. (March 2024)

Rybrevant Gets FDA Approval for EGFR Exon 20 Insertion-Mutated Non-Small Cell Lung Cancer Indications
The FDA approved Janssen Biotech’s Rybrevant (amivantamab-vmjw) with carboplatin and pemetrexed for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test.

Traditional approval was granted to Rybrevant for adults with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. FDA previously granted accelerated approval for this indication.

For this review, FDA collaborated with the Brazilian Health Regulatory Agency (ANVISA) and Health Canada. The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (March 2024)

FDA Approves osimertinib with chemotherapy for EGFR-mutated non-small cell lung cancer
AstraZeneca’s Tagrisso (osimertinib) was granted FDA approval with platinum-based chemotherapy for patients with locally advanced or metastatic non-small cell lung cancer (la/mNSCLC) whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test.

This application was granted priority review, fast track designation, breakthrough therapy designation, and orphan drug designation. (February 2024)

Amtagvi Gets FDA Accelerated Approval for Unresectable or Metastatic Melanoma
The FDA granted accelerated approval to Iovance Biotherapeutics’ Amtagvi (lifileucel), a tumor-derived autologous T cell immunotherapy, for adults with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 positive, a BRAF inhibitor with or without a MEK inhibitor.

This application was granted priority review, fast track designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation. (February 2024)

FDA Approves Tepmetko for Metastatic Non-Small Cell Lung Cancer
EMD Serono’s Tepmetko was granted FDA traditional approval for adults with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations.

Tepotinib was previously granted accelerated approval for this indication on February 3, 2021, based on initial overall response rate (ORR) and duration of response (DOR) in the VISION trial (NCT02864992), a multicenter, non-randomized, open-label, multicohort study. The conversion to traditional approval was based on an additional 161 patients and an added 28 months of follow-up time to assess DOR. This application was granted breakthrough designation and orphan drug designation. (February 2024)

Onivyde Gets FDA Approval for First-Line Treatment of Metastatic Pancreatic Adenocarcinoma
The FDA approved Ipsen’s Onivyde (irinotecan liposome) with oxaliplatin, fluorouracil, and leucovorin, for the first-line treatment of metastatic pancreatic adenocarcinoma.

This application was granted orphan drug designation. (February 2024)

Balversa Gains FDA Approval for Locally Advanced or Metastatic Urothelial Carcinoma
Janssen Biotech’s Balversa (erdafitinib) gets approval from the FDA for adults with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy. Balversa is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy.

This approval amends the indication previously granted under accelerated approval for patients with mUC with susceptible FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy. This application was granted priority review. (January 2024)

FDA Approves Keytruda with Chemoradiotherapy for FIGO 2014 Stage III-IVA Cervical Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) with chemoradiotherapy (CRT) for people with FIGO 2014 Stage III-IVA cervical cancer.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA) and the Brazilian Health Regulatory Agency (ANVISA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (January 2024)

Padcev Gets FDA Approval with Keytruda for Locally Advanced or Metastatic Urothelial Cancer
Astellas’ Padcev (enfortumab vedotin-ejfv) in combination with Merck’s Keytruda (pembrolizumab) received FDA approval for people with locally advanced or metastatic urothelial cancer (la/mUC). FDA previously granted accelerated approval to this combination for people with la/mUC who are ineligible for cisplatin-containing chemotherapy.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration and Health Canada. This application was granted priority review and breakthrough designation. (December 2023)

Welireg Receives FDA Approval for Advanced Renal Cell Carcinoma
The FDA approved Merck’s Welireg (belzutifan) for people with advanced renal cell carcinoma (RCC) following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI). This application was granted priority review. (December 2023)

FDA Approves IWILFIN for High-Risk Neuroblastoma
USWM receives FDA approval for IWILFIN (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.

This application was granted priority review, breakthrough designation, and orphan drug designation. (December 2023)

Jaypirca Gets Accelerated Approval to Jaypirca for Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma
The FDA granted accelerated approval to Eli Lilly for Jaypirca (pirtobrutinib) for adults with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor. This application was granted priority review and orphan drug designation. (December 2023)

FDA Approves OGSIVEO for First Treatment for Desmoid Tumors
SpringWorks Therapeutics’ OGSIVEO (nirogacestat) receives FDA approval for adults with progressing desmoid tumors who require systemic treatment. This application was granted priority review, breakthrough designation, fast track designation, and orphan drug designation. (November 2023)

Xtandi Gains FDA Approval for Non-Metastatic Castration-Sensitive Prostate Cancer with Biochemical Recurrence
The FDA approved Astellas’ Xtandi (enzalutamide) for non-metastatic castration-sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastasis (high-risk BCR).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted priority review and Fast Track Designation. (November 2023)

FDA Approves Truqap with Fulvestrant for Breast Cancer
AstraZeneca’s Truqap (capivasertib) was gains FDA approval with fulvestrant for adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations, as detected by an FDA-approved test, following progression on at least one endocrine-based regimen in the metastatic setting or recurrence on or within 12 months of completing adjuvant therapy.

FDA also approved the FoundationOne®CDx assay as a companion diagnostic device to identify patients with breast cancer for treatment with capivasertib with fulvestrant.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Israel’s Ministry of Health (IMoH), Singapore’s Health Sciences Authority (HSA), Switzerland’s Swissmedic, and United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (November 2023)

Keytruda Gets FDA Approval with Chemotherapy for HER2-Negative Gastric or Gastroesophageal Junction Adenocarcinoma
The FDA approved pembrolizumab (Keytruda, Merck) with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted orphan drug designation. (November 2023)

FDA Approves Augtyro for ROS1-Positive Non-Small Cell Lung Cancer
Bristol-Myers Squibb’s Augtyro (repotrectinib) gets FDA approval for locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).

This is the first FDA approval that includes patients with ROS1-positive NSCLC who have previously received a ROS1 tyrosine kinase inhibitor (TKI), in addition to patients who are TKI naïve. This application was granted priority review, breakthrough designation, and fast track designation. (November 2023)

Fruzaqla Gets FDA Approval for Refractory Metastatic Colorectal Cancer
Takeda Pharmaceuticals’ Fruzaqla (fruquintinib) receives FDA approval for adults with metastatic colorectal cancer (mCRC) who received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Singapore’s Health Sciences Authority (HSA), Switzerland’s Swissmedic, and United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (November 2023)

FDA Amends Keytruda’s Gastric Cancer Indication
The FDA revised the existing indication of Merck’s Keytruda (pembrolizumab) with trastuzumab, fluoropyrimidine, and platinum-containing chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This updated indication, which remains approved under accelerated approval regulations, restricts its use to patients whose tumors express PD-L1 (CPS ≥ 1) as determined by an FDA-approved test.

The FDA also approved the Agilent PD-L1 IHC 22C3 pharmDx as a companion diagnostic device to select patients with gastric or GEJ adenocarcinoma whose tumors express PD-L1 (CPS ≥ 1). (November 2023)

Keytruda Granted FDA Approval with Chemotherapy for Biliary Tract Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) to be used with gemcitabine and cisplatin for locally advanced unresectable or metastatic biliary tract cancer (BTC).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. The application reviews are ongoing at the other regulatory agencies. (October 2023)

FDA Approves LOQTORZ for Nasopharyngeal Carcinoma
The FDA approved Coherus BioSciences’ LOQTORZ (toripalimab-tpzi) with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent, locally advanced nasopharyngeal carcinoma (NPC). FDA also approved toripalimab-tpzi as a single agent for adults with recurrent unresectable or metastatic NPC with disease progression on or after a platinum-containing chemotherapy. This application was granted priority review, breakthrough designation and orphan drug designation. (October 2023) 

Tibsovo Granted FDA Approval for Myelodysplastic Syndromes
The FDA approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) for adults with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, as detected by an FDA-approved test. The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic device to select people for ivosidenib. 

This application was granted priority review, breakthrough designation, and orphan drug designation. (October 2023)

FDA Expands Pediatric Indication for Rozlytrek and Approves New Pellet Formulation
Genentech’s Rozlytrek (entrectinib) was granted accelerated approval from the FDA for young children older than 1 month with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory standard therapy. In August 2019, FDA granted accelerated approval to entrectinib for people 12 years of age and older for this indication.

FDA also approved a new oral pellet formulation for entrectinib, and the prescribing information now includes instructions for making an oral suspension from the capsules.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. This application was granted priority review, breakthrough designation and orphan drug designation. (October 2023)

FDA Approves Neoadjuvant/ Adjuvant Keytruda for Resectable Non-Small Cell Lung Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) with platinum-containing chemotherapy as neoadjuvant treatment, and with continuation of single-agent pembrolizumab as post-surgical adjuvant treatment for resectable (tumors ≥4 cm or node positive) non-small cell lung cancer. (October 2023)

Opdivo Gets FDA Approval for Adjuvant Treatment of Stage IIB/C Melanoma
Bristol-Myers Squibb’s Opdivo (nivolumab) received FDA approval for the adjuvant treatment of completely resected Stage IIB/C melanoma in people 12 years and older.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Israel’s Ministry of Health (IMoH) and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted orphan drug designation. (October 2023)

FDA Approves Braftovi with Mektovi for Metastatic Non-Small Cell Lung Cancer with a BRAF V600E Mutation
The FDA approved Array BioPharma’s Braftovi (encorafenib) with Array BioPharma’s Mektovi (binimetinib) for adults with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, as detected by an FDA-approved test.

FDA also approved the FoundationOne CDx (tissue) and FoundationOne Liquid CDx (plasma) as companion diagnostics for encorafenib with binimetinib. If no mutation is detected in a plasma specimen, the tumor tissue should be tested. This application was granted orphan drug designation. (October 2023)

FDA Approves Bosulif for Children with Chronic Myelogenous Leukemia
Pfizer’s Bosulif (bosutinib) received FDA approval for children 1 year of age and older with chronic phase Ph+ chronic myelogenous leukemia that is newly diagnosed or resistant or intolerant to prior therapy. The FDA also approved a new capsule dosage form available in strengths of 50 mg and 100 mg. This application was granted priority review and orphan drug designation. (September 2023) 

Temodar Gains New and Updated Indications Under FDA Project Renewal Initiative
The FDA approved updated labeling for Merck’s Temodar (temozolomide) under Project Renewal, an Oncology Center of Excellence (OCE) initiative aimed at updating labeling information for older oncology drugs to ensure information is clinically meaningful and scientifically up-to-date. This is the second drug to receive a labeling update under this pilot program. 

Temodar is now approved for the following new and revised indications:

• adjuvant treatment of adults with newly diagnosed anaplastic astrocytoma.
• treatment of adults with refractory anaplastic astrocytoma.

One approved indication for Temodar remains the same:

• treatment of adults with newly diagnosed glioblastoma, concomitantly with radiotherapy and then as maintenance treatment.

Labeling for the dosage regimen was revised and updated for newly diagnosed glioblastoma and refractory anaplastic astrocytoma.

Project Renewal is limited to updating labeling of older oncology drugs with decades of use, multiple supportive clinical studies, and substantial post-marketing experience. (September 2023)

HEPZATO KIT Receives FDA Approval as a Liver-Directed Treatment for Uveal Melanoma
Delcath Systems’ HEPZATO KIT (melphalan for Injection/Hepatic Delivery System) received the Food and Drug Administration approval containing melphalan as a liver-directed treatment for adults with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.

Melphalan is administered via the device constituent part, Hepatic Delivery System (HDS), by infusion into the hepatic artery every 6 to 8 weeks for up to 6 total infusions. The recommended melphalan dose is 3 mg/kg based on ideal body weight, with a maximum dose of 220 mg during a single treatment.This application was granted orphan drug designation. (August 2023)

FDA Grants Accelerated Approval to Elrexfio for Multiple Myeloma
The FDA granted accelerated approval to Pfizer’s Elrexfio (elranatamab-bcmm), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, and Switzerland’s Swissmedic.The application reviews are ongoing at the other regulatory agencies.This application was granted priority review, breakthrough designation and orphan drug designation. (August 2023).

Akeega Plus Prednisone Receives FDA Approval for BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer
The FDA approved the fixed dose combination of Janssen Biotech’s Akeega (niraparib and abiraterone acetate) with prednisone, for adults with deleterious or suspected deleterious BRCA-mutated castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved test.

Patients receiving niraparib and abiraterone acetate plus prednisone should also receive a GnRH analog concurrently or should have had bilateral orchiectomy. This application was granted priority review. (August 2023)

FDA Grants Accelerated Approval to Talvey for Relapsed or Refractory Multiple Myeloma
Janssen Biotech’s Talvey (talquetamab-tgvs) was granted FDA accelerated approval for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA) and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies.This application was granted priority review, breakthrough designation, and orphan drug designation.  (August 2023)

Gavreto Granted FDA Regular Approval for Non-Small Cell Lung Cancer with RET Gene Fusions
The FDA granted regular approval to Genentech’s Gavreto (pralsetinib) for adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test.

Pralsetinib was previously granted accelerated approval for the NSCLC indication in September 2020, based on initial overall response rate (ORR) and duration of response (DOR) in 114 patients enrolled in the ARROW trial (NCT03037385), a multicenter, open-label, multi-cohort trial. The conversion to regular approval was based on data from an additional 123 patients and 25 months of additional follow-up to assess durability of response. This application was granted breakthrough designation and orphan drug designation. (August 2023) 

FDA Approves Lonsurf with Bevacizumab for Previously Treated Metastatic Colorectal Cancer
Taiho Oncology’s Lonsurf (trifluridine and tipiracil) receives FDA approval with bevacizumab, for metastatic colorectal cancer (mCRC) previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy. FDA had previously approved single-agent LONSURF for this indication in September 2015. This application was granted priority review designation.  (August 2023)

Jemperli Receives FDA Approval with Chemotherapy for Endometrial Cancer
The FDA approved GlaxoSmithKline’s Jemperli (dostarlimab-gxly) with carboplatin and paclitaxel, followed by single-agent dostarlimab-gxly, for primary advanced or recurrent endometrial cancer (EC) that is mismatch repair deficient (dMMR), as determined by an FDA-approved test, or microsatellite instability-high (MSI-H).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Switzerland’s Swissmedic (SMC), and United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review and breakthrough designation. (July 2023)

FDA Approves Vanflyta for Newly Diagnosed Acute Myeloid Leukemia
Daiichi Sankyo’s received approval from the FDA for Vanflyta (quizartinib) with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) that is FLT3 internal tandem duplication (ITD)-positive, as detected by an FDA-approved test. FDA also approved LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic for Vanflyta. Quizartinib is not indicated as maintenance monotherapy following allogeneic HSCT. This application was granted priority review, fast track designation and orphan drug designation. (July 2023)

Talzenna Receives FDA Approval with Enzalutamide for HRR Gene-Mutated Metastatic Castration-Resistant Prostate Cancer
The FDA approved Pfizer’s Talzenna (talazopari) with enzalutamide for homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC). Men receiving talazoparib and enzalutamide should also receive a GnRH analog concurrently or should have had bilateral orchiectomy. This application was granted priority review. (June 2023)

FDA Grants Accelerated Approval to Columvi for Selected Relapsed or Refractory Large B-Cell Lymphomas
Genentech’s Columvi (glofitamab-gxbm) was granted accelerated approval by the FDA for relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified (DLBCL, NOS) or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after two or more lines of systemic therapy. This application was granted priority review and fast track designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. (June 2023)

Lynparza Receives FDA Approval with Abiraterone and Prednisone (or Prednisolone) for BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer
The FDA approved AstraZeneca’s Lynparza (olaparib) with abiraterone and prednisone (or prednisolone) for adults with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved companion diagnostic test. (May 2023)

Epkinly Receives FDA Accelerated Approval for Relapsed or Refractory Diffuse Large B-Cell Lymphoma and High-Grade B-Cell Lymphoma
The FDA granted accelerated approval for Genmab’s Epkinly (epcoritamab-bysp) for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after two or more lines of systemic therapy. This application was granted priority review. (May 2023)

FDA Approves Polivy for Previously Untreated Diffuse Large B-Cell Lymphoma, Not Otherwise Specified, and High-Grade B-Cell Lymphoma
The FDA approved Genentech’s Polivy (polatuzumab vedotin-piiq) with a rituximab product, cyclophosphamide, doxorubicin, and prednisone (R-CHP) for adults who have previously untreated diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS), or high-grade B-cell lymphoma (HGBL) and who have an International Prognostic Index (IPI) score of 2 or greater. This application was granted orphan drug designation. (April 2023)

Omisirge Receives FDA Approval for People with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation
Gamida Cell Ltd received the FDA approval for Omisirge (omidubicel-onlv), a substantially modified allogeneic (donor) cord blood-based cell therapy to quicken the recovery of neutrophils (a subset of white blood cells) in the body and reduce the risk of infection. The product is intended for use in adults and children 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen (treatment such as radiation or chemotherapy).

“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers.”

Blood cancers are a form of cancer caused by uncontrolled growth of cells in the blood, disrupting the ability of blood cells to perform their normal functions. This abnormal cell growth often begins in the bone marrow, which is made up of stem cells that form into different types of blood cells with specific functions in the body. Blood cancers represent about 10% of all cases of cancer each year in the U.S. Blood cancers can be fatal, with varying survival rates based on multiple factors including the specific type of blood cancer diagnosed. This type of cancer can also cause serious and damaging effects to the body and lead to symptoms such as fatigue, bone and joint pain, night sweats, infections, weakness, weight loss and fever.

Stem cell transplantation is a common treatment for blood cancers. It involves putting healthy stem cells into the body to help restore the normal production and function of blood cells. One source of healthy stem cells is umbilical cord blood. Generally, before receiving this kind of transplant, the patient will undergo a course of treatments to remove their own stem cells and prepare the body for the new stem cells. This process may include undergoing therapies such as radiation or chemotherapy, both of which may weaken an individual’s immune system. As a result, a frequent and serious risk of this treatment is the occurrence of severe and sometimes deadly infections.

Omisirge, administered as a single intravenous dose, is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide (a form of vitamin B3). Each dose is patient-specific, containing healthy stem cells from an allogeneic pre-screened donor, meaning it comes from a different individual rather than using the patient’s own cells.

This application received Priority Review, Breakthrough Therapy and Orphan designations. (April 2023)

FDA Grants Accelerated Approval to Padcev with Keytruda for Locally Advanced or Metastatic Urothelial Carcinoma
The FDA granted accelerated approval to Astellas Pharma for Padcev (enfortumab vedotin-ejfv) with Merck’s Keytruda (pembrolizumab) for people with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy. This application was granted priority review and breakthrough designation. (April 2023)

Zynyz Gets FDA Accelerated Approval for Metastatic or Recurrent Locally Advanced Merkel Cell Carcinoma
Incyte receives accelerated approval from the FDA for Zynyz (retifanlimab-dlwr) for adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC).

MCC is a rare and aggressive type of skin cancer that frequently appears as a single, painless, reddish-purple skin nodule on the head, neck and arms in skin exposed to sunlight. MCC tends to grow quickly and has a high rate of metastatic disease, leading to a poor prognosis. MCC impacts less than 1 per 100,000 people in the U.S., but incidence rates are rapidly rising, especially in adults over the age of 65.

“More than a third of patients with MCC present with regional or distant metastases, which are associated with high rates of mortality,” said Dr. Shailender Bhatia, University of Washington and Fred Hutchinson Cancer Center. “The approval of Zynyz offers healthcare providers another first-line treatment option against MCC that can result in durable responses in patients with metastatic disease, and I look forward to having Zynyz in our treatment portfolio for these difficult-to-treat patients.”

This application was granted priority review, fast track, and orphan drug designation. (March 2023)

FDA Approves Tafinlar with Mekinist for Children with Low-Grade Glioma with a BRAF V600E Mutation
The FDA approved Novartis’ Tafinlar (dabrafenib) with Novartis’ Mekinist (trametinib) for children 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. The FDA also approved new oral formulations of both drugs suitable for patients who cannot swallow pills.

This represents the first FDA approval of a systemic therapy for the first-line treatment of pediatric patients with LGG with a BRAF V600E mutation. This application was granted priority review, breakthrough designation and orphan drug designation. (March 2023)

Verzenio Gets Expanded FDA Early Breast Cancer Indication with Endocrine Therapy
The FDA approved Eli Lilly and Company’s Verzenio (abemaciclib) with endocrine therapy (tamoxifen or an aromatase inhibitor) for the adjuvant treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, node-positive, early breast cancer at high risk of recurrence.

People defined as high risk included those having either ≥4 pALN (pathologic axillary lymph nodes) or 1-3 pALN and either tumor grade 3 or a tumor size ≥50 mm. Abemaciclib was previously approved for the above high-risk population with the additional requirement of having a Ki-67 score ≥20%. The approval removes the Ki-67 testing requirement.This application was granted priority review. (March 2023)

FDA Grants Regular Approval to Jemperli for dMMR Endometrial Cancer
GlaxoSmithKline received approval from the FDA for Jemperli (dostarlimab-gxly) for adults with mismatch repair deficient (dMMR) recurrent or advanced endometrial cancer, as determined by an FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation.

In April 2021, Jemperli received accelerated approval for adult patients with dMMR recurrent or advanced endometrial cancer, as determined by an FDA-approved test, that has progressed on or following a prior platinum-containing regimen. (February 2023)

Trodelvy Receives FDA Approval for HR-Positive Breast Cancer
The FDA approved Gilead Sciences’ Trodelvy (sacituzumab govitecan-hziy) for people with unresectable locally advanced or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (February 2023)

Orserdu Gets FDA Approval for ER-Positive, HER2-Negative, ESR1-Mutated Advanced or Metastatic Breast Cancer
The FDA approved Stemline Therapeutics’ Orserdu (elacestrant) for postmenopausal women or adult men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

FDA also approved the Guardant360 CDx assay as a companion diagnostic device to identify patients with breast cancer for treatment with elacestrant. This application was granted priority review and fast track designation. (January 2023)

FDA Grants Accelerated Approval to Jaypirca for Relapsed or Refractory Mantle Cell Lymphoma
Eli Lilly and Company was granted FDA accelerated approval for Jaypirca (pirtobrutinib) for relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor.

This application was granted priority review and fast track designation. The application also was granted orphan drug designation. (January 2023) 

Keytruda Gets FDA Approval as Adjuvant Treatment for Non-Small Cell Lung Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) for adjuvant treatment following resection and platinum-based chemotherapy for stage IB (T2a ≥4 cm), II, or IIIA non-small cell lung cancer (NSCLC).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies.  (January 2023)

FDA Grants Accelerated Approval to Tukysa with Trastuzumab for Colorectal Cancer
The FDA granted accelerated approval to Seagen for Tukysa (tucatinib) in combination with trastuzumab for RAS wild-type HER2-positive unresectable or metastatic colorectal cancer that has progressed following fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA). The application review is ongoing at the other regulatory agency.This application was granted priority review and breakthrough therapy designation. Tukysa was granted orphan drug designation for the treatment of HER2+ colorectal cancer. (January 2023)

Brukinsa Gets FDA Approval for Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
BeiGene received approval from the FDA for Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). This application was granted orphan drug designation. (January 2023)

Lunsumio Receives FDA Accelerated Approval For Relapsed or Refractory Follicular Lymphoma
The FDA granted Genentech accelerated approval for Lunsumio (mosunetuzumab-axgb), a bispecific CD20-directed CD3 T-cell engager for adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This application was granted priority review and breakthrough designation. The application also was granted orphan drug designation. (December 2022)

FDA Approves First Adenoviral Vector-Based Gene Therapy for High-Risk Bacillus Calmette-Guérin Unresponsive Non-Muscle Invasive Bladder Cancer
Ferring Pharmaceuticals received approval from the FDA for Adstiladrin (nadofaragene firadenovec-vncg) for adults with high-risk Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. This application was granted fast-track, breakthrough therapy, and orphan drug designation. (December 2022)

FDA Grants Accelerated Approval to Krazati for KRAS G12C-Mutated NSCLC
The FDA granted accelerated approval to Mirati Therapeutics’ Krazati (adagrasib), a RAS GTPase family inhibitor, for adults with KRAS G12C¬-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.

FDA also approved the QIAGEN therascreen KRAS RGQ PCR kit (tissue) and the Agilent Resolution ctDx FIRST Assay (plasma) as companion diagnostics for Krazati. If no mutation is detected in a plasma specimen, the tumor tissue should be tested.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). This application was granted fast-track, breakthrough therapy, and orphan drug designation. (December 2022)

Tecentriq Gets FDA Approval for Alveolar Soft Part Sarcoma
Genentech received approval from the FDA for Tecentriq (atezolizumab) for adults and children 2 years of age and older with unresectable or metastatic alveolar soft part sarcoma (ASPS). This application was granted priority review and breakthrough designation. The application also was granted orphan drug designation. (December 2022)

FDA Approves Rezlidhia for Relapsed or Refractory Acute Myeloid Leukemia with a Susceptible IDH1 Mutation
The FDA approved Rigel Pharmaceuticals’ Rezlidhia (olutasidenib) capsules for people with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. The FDA also approved the Abbott RealTime IDH1 Assay to select patients for olutasidenib. (December 2022) 

FDA Grants Accelerated Approval to Elahere for FRα Positive, Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Peritoneal Cancer
ImmunoGen received FDA accelerated approval for Elahere (mirvetuximab soravtansine-gynx) for adults with folate receptor alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Mirvetuximab soravtansine-gynx is a folate receptor alpha directed antibody and microtubule inhibitor conjugate. People are selected for therapy based on an FDA-approved test.

The FDA also approved Ventana Medical Systems’ VENTANA FOLR1 (FOLR-2.1) RxDx Assay as a companion diagnostic device to select adults for the above indication. This application was granted priority review. (November 2022) 

Imjudo Gets FDA Approval in Combination with Imfinzi and Platinum-Based Chemotherapy for Metastatic Non-Small Cell Lung Cancer
AstraZeneca Pharmaceuticals received FDA approval for Imjudo (tremelimumab) in combination with Imfinzi and platinum-based chemotherapy for adult patients with metastatic non-small cell lung cancer (NSCLC) with no sensitizing epidermal growth factor receptor (EGFR) mutation or anaplastic lymphoma kinase (ALK) genomic tumor aberrations. (November 2022)

FDA Approves Adcetris in Combination with Chemotherapy for Pediatric Patients with Classical Hodgkin Lymphoma
The FDA approved Seagen’s Adcetris (brentuximab vedotin) in combination with doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide for pediatric patients 2 years of age and older with previously untreated high risk classical Hodgkin lymphoma (cHL). This is the first pediatric approval for brentuximab vedotin. This application was granted priority review. Brentuximab vedotin has orphan drug designation for the treatment of Hodgkin lymphoma. (November 2022)

Libtayo Receives FDA approval in Combination with Platinum-Based Chemotherapy for Non-Small Cell Lung Cancer
Regeneron Pharmaceuticals receives FDA approval for Libtayo (cemiplimab-rwlc) in combination with platinum-based chemotherapy for adults with advanced non-small cell lung cancer (NSCLC) with no EGFR, ALK, or ROS1 aberrations. (November 2022)

FDA Approves Tecvayli for Relapsed or Refractory Multiple Myeloma
The FDA granted accelerated approval to Janssen Biotech’s Tecvayli (teclistamab-cqyv), the first bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies.

This application was granted priority review and breakthrough designation. The application also was granted orphan drug designation. (October 2022)

Imjudo Receives FDA Approval in Combination with Durvalumab for Unresectable Hepatocellular Carcinoma
AstraZeneca Pharmaceuticals received approval from the FDA for Imjudo (tremelimumab) in combination with durvalumab for adults with unresectable hepatocellular carcinoma (uHCC). Tremelimumab was granted orphan drug designation. (October 2022)

FDA Grants Accelerated Approval to Lytgobi for Cholangiocarcinoma
The FDA granted accelerated approval to Taiho Oncology’s Lytgobi (futibatinib) for adults with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. This application was granted priority review and breakthrough designation. The application also was granted orphan drug designation. (September 2022) 

Retevmo Granted FDA Regular Approval for Locally Advanced or Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer
The FDA granted regular approval to Eli Lilly and Company’s Retevmo (selpercatinib) for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a rearranged during transfection (RET) gene fusion, as detected by an FDA-approved test.

The FDA also approved the Oncomine Dx Target (ODxT) Test (Thermo Fisher Scientific) as a companion diagnostic for selpercatinib.

Selpercatinib was previously granted accelerated approval for the NSCLC indication in 2020. The conversion to regular approval was based on data from an additional 172 patients and 18 months of additional follow-up to assess durability of response.

This application was granted breakthrough designation. The application also was granted orphan drug designation. (September 2022)

Retevmo Receives FDA Approval for Locally Advanced or Metastatic RET Fusion-Positive Solid Tumors
Eli Lilly and Company was granted FDA accelerated approval for Retevmo (selpercatinib) for adults with locally advanced or metastatic solid tumors with a rearranged during transfection (RET) gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options.

This application was granted accelerated approval based on overall response rate and duration of response. Continued approval may be contingent upon verification of clinical benefit in confirmatory trials. This application was granted priority review and was granted orphan drug designation. (September 2022)

FDA Approves Pedmark to Reduce the Risk of Ototoxicity Associated with Cisplatin in Children with Localized, Non-Metastatic Solid Tumor
The FDA approved Fennec Pharmacueticals’ Pedmark (sodium thiosulfate) to reduce the risk of ototoxicity associated with cisplatin in children 1 month and older with localized, non-metastatic solid tumors. The application also was granted Orphan Drug designation. (September 2022) 

FDA Approves Imfinzi for Locally Advanced or Metastatic Biliary Tract Cancer
AstraZeneca receives FDA approval for Imfinzi (durvalumab) in combination with gemcitabine and cisplatin for adults with locally advanced or metastatic biliary tract cancer (BTC).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Singapore’s Health Sciences Authority (HSA), and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies.

This application was granted priority review and was granted orphan drug designation. (September 2022)

Pemazyre Receives FDA Approval for Relapsed or Refractory Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement
Incyte Corporation receives FDA approval for Pemazyre (pemigatinib) for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement. This application was granted priority review and breakthrough designation and granted orphan drug designation. (August 2022)

FDA Approves Imbruvica for Children with Chronic Graft Versus Host Disease, Including a New Oral Suspension
The FDA approves Pharmacyclics’ Imbruvica (ibrutinib) for children ≥ 1 year of age with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy. Formulations include capsules, tablets, and oral suspension. The applications for this action were granted priority review. Ibrutinib also was granted orphan drug designation. (August 2022) 

Enhertu Gets FDA Accelerated Approval for HER2-Mutant Non-Small Cell Lung Cancer
The FDA granted accelerated approval to Daiichi Sankyo’s Enhertu (fam-trastuzumab deruxtecan-nxki) for adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating human epidermal growth factor receptor 2 HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. This is the first drug approved for HER2-mutant NSCLC.

FDA also approved the Life Technologies Corporation’s Oncomine™ Dx Target Test (tissue) and the Guardant Health, Inc.’s Guardant360® CDx (plasma) as companion diagnostics for Enhertu. If no mutation is detected in a plasma specimen, the tumor tissue should be tested.

This application used advice from the FDA Oncology Center of Excellence Project Optimus to conduct a dose randomization study, which led to a lower dose being approved. This application was granted priority review and breakthrough designation. (August 2022)

FDA Approves Tabrecta for Metastatic Non-Small Cell Lung Cancer
Novartis Pharmaceuticals was granted FDA regular approval for Tabrecta (capmatinib) for adults with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation leading to mesenchymal-epithelial transition (MET) exon 14 skipping, as detected by an FDA-approved test.

Capmatinib was previously granted accelerated approval for the same indication in 2020, based on initial overall response rate and duration of response in the GEOMETRY mono-1 trial (NCT02414139), a multicenter, non-randomized, open-label, multi-cohort study. The conversion to regular approval was based on data from an additional 63 patients, as well as an additional 22 months of follow- up time to assess durability of response and verify clinical benefit.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA). The application reviews may be ongoing at the other regulatory agencies.

This application was granted priority review and breakthrough designation and was granted orphan drug designation. (August 2022) 

Calquence Tablet Formulation Receives Approval Across Current Indications
The FDA has approved AstraZeneca’s Calquence (acalabrutinib) new tablet formulation for all current indications, including adults with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL) and for people with relapsed or refractory mantle cell lymphoma (MCL), which is approved under accelerated approval based on overall response rate. The new formulation offers equivalent efficacy, safety and consistent dosing compared to the current capsule. The tablet can be taken with gastric acid-reducing agents, including proton pump inhibitors (PPIs), antacids and H2-receptor antagonists (H2RAs).

John C. Byrd, MD, Chair of the Department of Internal Medicine at the University of Cincinnati, said: “Patients with blood cancers like chronic lymphocytic leukemia and mantle cell lymphoma are often older and may face multiple medical conditions that may need intervention, including acid reflux or peptic ulcer disease. The US approval of acalabrutinib in a tablet form enables co-administration of the acalabrutinib tablet alongside a proton pump inhibitor. This provides another option for some patients with chronic lymphocytic leukemia and relapsed or refractory mantle cell lymphoma, enabling more patients to potentially benefit from this treatment.”

Calquence is also approved as a capsule formulation for the same indications as the tablet in the US and in many other countries worldwide. (August 2022)

FDA Approves First Targeted Therapy for HER2-Low Breast Cancer
The FDA approved Daiichi Sankyo’s Enhertu (fam-trastuzumab-deruxtecan-nxki), an IV infusion for the treatment of people with unresectable (unable to be removed) or metastatic (spread to other parts of the body) HER2-low breast cancer. This is the first approved therapy targeted to patients with the HER2-low breast cancer subtype, which is a newly defined subset of HER2-negative breast cancer.

It is estimated that 287,850 new cases of female breast cancer will be diagnosed in 2022 in the U.S. Approximately 80-85% of those new cases were previously considered to be HER2-negative subtype (including hormone receptor positive and triple negative breast cancer), which means the tumors do not overexpress, or make too many copies of the HER2 protein. Of that proportion of breast cancer diagnoses, about 60% of patients previously classified as having HER2-negative subtype can now be considered as HER2-low. Prior to this approval, HER2-low patients received endocrine therapy or chemotherapy.

“Having therapies that are specially tailored to each patient’s cancer subtype is a priority to ensure access to safe and innovative treatments” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

HER2 receptors, which are proteins made by the HER2 gene, are important in determining a patient’s treatment. HER2-negative includes hormone receptor positive and triple negative breast cancers. HER2-low is a new classification of the HER2 subtype. It describes a new subtype of breast cancer that has some HER2 proteins on the cell surface, but not enough to be classified as HER2-positive.

People with HER2-low breast cancer are eligible for Enhertu if they have received a prior chemotherapy in the metastatic setting, or their cancer returned during, or within 6 months of completing, adjuvant chemotherapy. Enhertu received priority review and breakthrough therapy designations for this indication. (August 2022)

Nubeqa Receives FDA Approval for Metastatic Hormone-Sensitive Prostate Cancer
Bayer HealthCare Pharmaceuticals received FDA approval for Nubeqa (darolutamide) tablets in combination with docetaxel for adults with metastatic hormone-sensitive prostate cancer (mHSPC).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Health Canada, the Singapore Health Sciences Authority, Switzerland’s Swissmedic, the United Kingdom’s Medicines and Healthcare products Regulatory Authority, and Australia’s Therapeutic Goods Administration. The application reviews may be ongoing at the other regulatory agencies. (August 2022)

FDA Approves Xalkori for ALK-Positive Inflammatory Myofibroblastic Tumor
Pfizer’s Xalkori (crizotinib) received FDA approval for adults and children 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT). This application was granted priority review and received orphan drug designation. (July 2022)

Breyanzi Gets FDA Approval for Second-line Treatment of Large B-Cell Lymphoma
Juno Therapeutics, Inc. received FDA approval for Breyanzi (lisocabtagene maraleucel) for adults with large B-cell lymphoma (LBCL) who have refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy; or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation (HSCT) due to comorbidities or age. It is not indicated for the treatment of patients with primary central nervous system lymphoma. This application was granted priority review, regenerative medicine advanced therapy designation, and breakthrough designation.  (June 2022)

FDA Grants Accelerated Approval to Tafinlar in Combination with Mekinist for Unresectable or Metastatic Solid Tumors with BRAF V600E Mutation
The FDA granted accelerated approval to Novartis’ Tafinlar (dabrafenib) in combination with Mekinist (trametinib) for the treatment of adults and pediatric children ≥ 6 years of age with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. Dabrafenib in combination with trametinib is not indicated for people with colorectal cancer because of known intrinsic resistance to BRAF inhibition. Dabrafenib is not indicated for people with wild-type BRAF solid tumors. This application was granted priority review. (June 2022)

FDA Approves Kymriah for Relapsed or Refractory Follicular Lymphoma
Novartis’ gets FDA accelerated approval for Kymriah (tisagenlecleucel) for adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). This application was granted priority review and regenerative medicine advanced therapy designation. The application also was granted orphan drug designation. (May 2022)

Opdivo Gets FDA Approval in Combination with Chemotherapy and Opdivo in Combination with Yervoy for First-Line Esophageal Squamous Cell Carcinoma Indications
The FDA approved the following for the first-line treatment of people with advanced or metastatic esophageal squamous cell carcinoma (ESCC):

• Bristol-Myers Squibb’s Opdivo (nivolumab) in combination with fluoropyrimidine- and platinum-based chemotherapy
• Opdivo in combination with Yervoy (ipilimumab)

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration, Health Canada, Israel’s Ministry of Health Pharmaceutical Administration, and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies. Opdivo was granted orphan drug designation for esophageal cancer. (May 2022)

Tibsovo Gets Approval in Combination with Azacitidine for Newly Diagnosed Acute Myeloid Leukemia
The FDA grants Servier Pharmaceuticals’ approval for Tibsovo (ivosidenib) in combination with azacitidine for newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. This application received orphan drug designation for AML on June 9, 2015. (May 2022)

FDA Approves Vidaza for Newly Diagnosed Juvenile Myelomonocytic Leukemia
Celgene receives FDA approval for Vidaza (azacytidine) for children with newly diagnosed juvenile myelomonocytic leukemia (JMML). This application was granted priority review, breakthrough designation. (May 2022)

FDA Grants Regular Approval for Enhertu for Breast Cancer
Daiichi Sankyo’s Enhertu (fam-trastuzumab deruxtecan-nxki) received FDA approval for adults with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within 6 months of completing therapy.

In December 2019, Enhertu received accelerated approval for adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting. The following trial was the confirmatory trial for the accelerated approval.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration, Health Canada, Israel’s Ministry of Health Pharmaceutical Administration, and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies. This application was granted priority review, breakthrough designation. (May 2022)

Vijoice Receives FDA Approval for PIK3CA-Related Overgrowth Spectrum
The FDA granted accelerated approval to Novartis’ Vijoice (alpelisib) for adult and children two years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. This application was granted priority review, breakthrough designation and orphan drug designation. (April 2022)

FDA Approves Yescarta for Second-Line Treatment of Large B-Cell Lymphoma
Kite Pharma received approval for Yescarta (axicabtagene ciloleucel) for adults with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line chemoimmunotherapy. It is not indicated for the treatment of people with primary central nervous system lymphoma. This application was granted priority review, breakthrough therapy designation, and orphan drug designation. (April 2022)

Pluvicto Gets FDA Approval for Metastatic Castration-Resistant Prostate Cancer
Advanced Accelerator Applications’ Pluvicto (lutetium Lu 177 vipivotide tetraxetan) received FDA approval for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor (AR) pathway inhibition and taxane-based chemotherapy. 

On the same day, the FDA approved Locametz (gallium Ga 68 gozetotide), a radioactive diagnostic agent for positron emission tomography (PET) of PSMA-positive lesions, including selection of men with metastatic prostate cancer for whom lutetium Lu 177 vipivotide tetraxetan PSMA-directed therapy is indicated. Locametz is the first radioactive diagnostic agent approved for patient selection in the use of a radioligand therapeutic agent. 

Patients with previously treated mCRPC should be selected for treatment with Pluvicto using Locametz or another approved PSMA-11 imaging agent based on PSMA expression in tumors. PSMA-positive mCRPC was defined as having at least one tumor lesion with gallium Ga 68 gozetotide uptake greater than normal liver. This application was granted priority review and breakthrough designation. (March 2022)

FDA Approves Keytruda for Advanced Endometrial Carcinoma
Merck’s Keytruda (pembrolizumab) received FDA approval as a single agent, for women with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and who are not candidates for curative surgery or radiation. 

The FDA also approved Ventana Medical Systems/Roche Tissue Diagnostics’ VENTANA MMR RxDx Panel as a companion diagnostic device to select people with dMMR in solid tumors that are eligible for treatment with pembrolizumab. The FDA previously approved the FoundationOne CDx (F1CDx, Foundation Medicine, Inc.) as a companion diagnostic device to select patients with MSI-H in solid tumors that are eligible for treatment with pembrolizumab. (March 2022)

Opdualag Receives FDA Approval for Unresectable or Metastatic Melanoma
The FDA has approved Bristol-Myers Squibb’s Opdualag (nivolumab and relatlimab-rmbw) for adult and children 12 years of age or older with unresectable or metastatic melanoma. Opdualag is a fixed-dose combination of the LAG-3-blocking antibody relatlimab and the programmed death receptor-1 blocking antibody nivolumab.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies. This application was granted priority review, fast track designation, and orphan drug designation. (March 2022)

Lynparza Gains FDA Approval for Adjuvant Treatment of High-risk Early Breast Cancer
AstraZeneca’s Lynparza (olaparib) gains approvals from the FDA for the adjuvant treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer who have been treated with neoadjuvant or adjuvant chemotherapy. Patients must be selected for therapy based on an FDA-approved companion diagnostic for Lynparza.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Health Canada and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies. This application was also granted priority review. (March 2022)

The FDA Approved Neoadjuvant Opdivo and Platinum-Doublet Chemotherapy for Early-Stage Non-Small Cell Lung Cancer 
The FDA approved Bristol-Myers Squibb’s Opdivo (nivolumab) with platinum-doublet chemotherapy for adult patients with resectable non-small cell lung cancer (NSCLC) in the neoadjuvant setting. This represents the first FDA approval for neoadjuvant therapy for early-stage NSCLC.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration, Health Canada, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency. This application was also granted priority review. (March 2022)

Carvykti Gets Approval for Relapsed or Refractory Multiple Myeloma 
Janssen Biotech, Inc. receives FDA approval of CARVYKTI (ciltacabtagene autoleucel) for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and an anti-CD38 monoclonal antibody.

Carvykti is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose is customized using a patient’s own T-cells, which are collected and genetically modified, and infused back into the patient. This application was granted priority review, breakthrough designation and orphan drug designation. (February 2022)

FDA Approves Kimmtrak for Unresectable or Metastatic Uveal Melanoma
Immunocore Limited received FDA approval for Kimmtrak (tebentafusp-tebn), a bispecific gp100 peptide-HLA-directed CD3 T cell engager, for HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma. Kimmtrak is the first and only FDA approved therapy for the treatment of unresectable or metastatic uveal melanoma. This application was granted priority review, breakthrough designation and orphan drug designation.

Uveal melanoma is a devastating disease that has historically resulted in death within a year of metastasis for our patients,” said John Kirkwood, MD, director of the Melanoma Center at the UPMC Hillman Cancer Center. “The approval of Kimmtrak represents a major paradigm shift in the treatment of metastatic uveal melanoma, and for the first time offers hope to those with this aggressive form of cancer.” (January 2022)

First Drug to Prevent Graft Versus Host Disease Approved by FDA
The FDA approved Bristol Myers Squibb’s Orencia (abatacept) for the prophylaxis (prevention) of acute graft versus host disease (aGVHD), a condition that occurs when donor bone marrow or stem cells attack the graft recipient, in combination with certain immunosuppressants. Orencia may be used in adults and children two years of age or older undergoing hematopoietic stem cell transplantation (commonly known as bone marrow transplantation or stem cell transplantation) from an unrelated donor. This is the first FDA drug approval for aGVHD prevention.

“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”

Acute GVHD is a potentially fatal complication that can occur after stem cell transplantation when the donor’s immune cells (the graft) view the recipient’s body (the host) as foreign, and the donated cells attack the body. The chances of developing aGVHD increase when the donor and recipient are not related or are not a perfect match.

Orencia received Breakthrough, Orphan Drug and Priority Review designations for this indication. Development of this product was partially supported by the FDA’s Orphan Products Grants Program, which provides grants for clinical studies on safety and efficacy of products for use in rare diseases or conditions. Orencia was originally approved by the FDA in 2005 for the treatment of adult rheumatoid arthritis. Orencia is also approved for the treatment of polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, the FDA collaborated with Health Canada, Swissmedic and MOH (Israel’s Ministry of Health). The application reviews are ongoing at the other regulatory agencies. (December 2021)

Keytruda Gets FDA Approval for Adjuvant Treatment of Stage IIB or IIC Melanoma
Merck’s Keytruda (pembrolizumab) has received FDA approval for the adjuvant treatment of adults and children 12 years of age and older with stage IIB or IIC melanoma following complete resection. This application was granted priority review and orphan designation. (December 2021)

FDA Approves Rituxan Plus Chemotherapy for Pediatric Cancer Indications
FDA granted Genentech’s Rituxan (rituximab) in combination with chemotherapy for children 6 months to 18 years with previously untreated, advanced stage, CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL), Burkitt-like lymphoma (BLL), or mature B-cell acute leukemia (B-AL). This application was granted priority review. (December 2021)

FDA Approves Darzalex Faspro, Kyprolis, and Dexamethasone for Multiple Myeloma
The FDA approved Janssen Biotech’s Faspro (daratumumab + hyaluronidase-fihj) and Amgen’s Kyprolis (carfilzomib) plus dexamethasone for adults with relapsed or refractory multiple myeloma who have received 1 to 3 prior lines of therapy. (November 2021)

FDA Approves New Imaging Drug to Help Identify Ovarian Cancer Lesions
The FDA approved On Target Laboratories’ Cytalux (pafolacianine), an imaging drug intended to assist surgeons in identifying ovarian cancer lesions. The drug is designed to improve the ability to locate additional ovarian cancerous tissue that is normally difficult to detect during surgery. Cytalux is indicated for use in adult patients with ovarian cancer to help identify cancerous lesions during surgery. The drug is a diagnostic agent that is administered in the form of an intravenous injection prior to surgery.

“The FDA’s approval of Cytalux can help enhance the ability of surgeons to identify deadly ovarian tumors that may otherwise go undetected,” said Alex Gorovets, MD, deputy director of the Office of Specialty Medicine in the FDA’s Center for Drug Evaluation and Research. “By supplementing current methods of detecting ovarian cancer during surgery, Cytalux offers health care professionals an additional imaging approach for patients with ovarian cancer.” The FDA previously granted Cytalux orphan-drug, priority and fast track designations. (November 2021)

Fyarro Approved for Malignant Perivascular Epithelioid Cell Tumor
Aadi Bioscience received FDA approval for Fyarro (sirolimus protein-bound particles) for injectable suspension (albumin-bound) for adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). This application was granted priority review, fast track designation, breakthrough therapy designation, and orphan drug designation. (November 2021)

Keytruda Approved for Adjuvant Treatment of Renal Cell Carcinoma
Merck received approval for Keytruda (pembrolizumab) for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Swissmedic. The application reviews may be ongoing at the other regulatory agencies. This application was granted priority review. (November 2021)

FDA Approved Besremi for Rare Blood Disease
The FDA approved PharmaEssentia Corporation’s Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. The excess cells thicken the blood, slowing blood flow and increasing the chance of blood clots.

“Over 7,000 rare diseases affect more than 30 million people in the United States. Polycythemia vera affects approximately 6,200 Americans each year,” said Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “This action highlights the FDA’s commitment to helping make new treatments available to patients with rare diseases.”

Besremi is the first FDA-approved medication for polycythemia vera that patients can take regardless of their treatment history, and the first interferon therapy specifically approved for polycythemia vera. Besremi received orphan drug designation for this indication. Orphan drug designation provides incentives to assist and encourage drug development for rare diseases. (November 2021)

FDA Grants Accelerated Approval to Scemblix for Philadelphia Chromosome-Positive Chronic Myeloid Leukemia
The FDA has granted accelerated approval to Novartis’ Scemblix (asciminib) for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), and approved asciminib for adults with Ph+ CML in CP with the T315I mutation. This application was granted priority review, breakthrough designations, fast track designation, and orphan drug designation. (October 2021)

Tecentriq Approved as Adjuvant Treatment for Non-Small Cell Lung Cancer
Genentech received approval for Tecentriq (atezolizumab) for adjuvant treatment following resection and platinum-based chemotherapy in patients with stage II to IIIA non-small cell lung cancer (NSCLC) whose tumors have PD-L1 expression on ≥ 1% of tumor cells, as determined by an FDA-approved test. The FDA also approved the VENTANA PD-L1 (SP263) Assay (Ventana Medical Systems, Inc.) as a companion diagnostic device to select patients with NSCLC for adjuvant treatment with Tecentriq.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, Switzerland’s Swissmedic, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (October 2021)

FDA Approved Keytruda in Combination for The First-Line Treatment of Cervical Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) in combination with chemotherapy, with or without bevacizumab, for patients with persistent, recurrent or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1), as determined by an FDA-approved test. FDA also granted regular approval to pembrolizumab as a single agent for patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. In June 2018, FDA had granted accelerated approval to this indication with the companion diagnostic, Dako North America’s PD-L1 IHC 22C3 pharmDx.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies.This application was granted priority review designation. (October 2021)

Verzenio Granted Approval with Endocrine Therapy for Early Breast Cancer
Eli Lilly and Company’s Verzennio (abemaciclib) has been given FDA approval with endocrine therapy (tamoxifen or an aromatase inhibitor) for adjuvant treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, node-positive, early breast cancer at high risk of recurrence and a Ki-67 score ≥20%, as determined by an FDA approved test. This is the first CDK 4/6 inhibitor approved for adjuvant treatment of breast cancer. FDA also approved Dako Omnis’ Ki-67 IHC MIB-1 pharmDx assay, submitted by Agilent, Inc., as a companion diagnostic for selecting people for this indication. (October 2021)

FDA Approves Tecartus for Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia
The FDA approved Kite Pharma’s Tecartus (brexucabtagene autoleucel) for patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This application was granted priority review, breakthrough designation and orphan drug designation. (October 2021)

Jakafi Approved for Chronic Graft-Versus-Host Disease
Inctye’s Jakafi (ruxolitinib) has received approval for chronic graft-versus-host disease (cGVHD) after failure of one or two lines of systemic therapy in adults and children 12 years and older.

For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, Switzerland’s Swissmedic and the United Kingdom’s Medicines & Healthcare products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies. This application was granted priority review and orphan product designation. (September 2021)

FDA Grants Accelerated Approval to Tivdak for Recurrent or Metastatic Cervical Cancer
The FDA granted accelerated approval to Seagen’s Tivdak (tisotumab vedotin-tftv), a tissue factor-directed antibody and microtubule inhibitor conjugate, for adults with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. This application was granted priority review. (September 2021)

Cabometyx Approved for Differentiated Thyroid Cancer
The FDA approved Exelixis’ Cabometyx (cabozantinib) for adults and children 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior VEGFR-targeted therapy and who are ineligible or refractory to radioactive iodine. This application was granted priority review, breakthrough designation and orphan drug designation.(September 2021)

FDA Authorizes Software that Can Help Identify Prostate Cancer
AI was authorized by the FDA to market software to assist medical professionals who examine body tissues (pathologists) in the detection of areas that are suspicious for cancer as an adjunct (supplement) to the review of digitally-scanned slide images from prostate biopsies (tissue removed from the body). The software, called Paige Prostate, is the first artificial intelligence (AI)-based software designed to identify an area of interest on the prostate biopsy image with the highest likelihood of harboring cancer so it can be reviewed further by the pathologist if the area of concern has not been identified on initial review.

“Pathologists examine biopsies of tissue suspected for diseases, such as prostate cancer, every day. Identifying areas of concern on the biopsy image can help pathologists make a diagnosis that informs the appropriate treatment,” said Tim Stenzel, MD, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “The authorization of this AI-based software can help increase the number of identified prostate biopsy samples with cancerous tissue, which can ultimately save lives.” (September 2021)

FDA Grants Accelerated Approval to Exkivity for Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations
The FDA granted accelerated approval to Takeda’s Exkivity (mobocertinib) for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.

The FDA also approved Life Technologies Corporation’s Oncomine Dx Target Test as a companion diagnostic device to select patients with the above mutations for mobocertinib treatment.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). This application was granted priority review, breakthrough therapy designation and orphan drug designation. (September 2021)

Brukinsa Gets FDA Accelerated Approval for Marginal Zone Lymphoma
The FDA granted accelerated approval to BeiGene’s Brukinsa (zanubrutinib) for adults with relapsed or refractory marginal zone lymphoma who have received at least one anti-CD20-based regimen.

This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. This application was granted priority review, Fast Track Designation and Orphan Designation. (September 2021)

FDA Approves Brukinsa for Waldenström’s Macroglobulinemia
The FDA approved BeiGene’s Brukinsa (zanubrutinib) for adults with Waldenström’s macroglobulinemia (WM). Zanubrutinib was investigated in ASPEN (NCT03053440), a randomized, active control, open-label trial, comparing zanubrutinib and ibrutinib in patients with MYD88 L265P mutation (MYD88MUT) WM. This application was granted Fast Track designation and Orphan Designation. (September 2021)

Tibsovo Gets FDA Approval for Advanced or Metastatic Cholangiocarcinoma
The FDA approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) for adults with previously treated, locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

The FDA also approved Life Technologies Corporation’s Oncomine Dx Target Test as a companion diagnostic device to aid in selecting patients with cholangiocarcinoma for treatment with ivosidenib. This application was granted priority review, fast track designation, and orphan product designation. (August 2021)

FDA Approves First COVID-19 Vaccine
The FDA approved the first COVID-19 vaccine. The vaccine has been known as the Pfizer-BioNTech COVID-19 Vaccine, and will now be marketed as Comirnaty (koe-mir’-na-tee), for the prevention of COVID-19 disease in individuals 16 years of age and older. The vaccine also continues to be available under emergency use authorization (EUA), including for individuals 12 through 15 years of age and for the administration of a third dose in certain immunocompromised individuals.

“The FDA’s approval of this vaccine is a milestone as we continue to battle the COVID-19 pandemic. While this and other vaccines have met the FDA’s rigorous, scientific standards for emergency use authorization, as the first FDA-approved COVID-19 vaccine, the public can be very confident that this vaccine meets the high standards for safety, effectiveness, and manufacturing quality the FDA requires of an approved product,” said Acting FDA Commissioner Janet Woodcock, MD. “While millions of people have already safely received COVID-19 vaccines, we recognize that for some, the FDA approval of a vaccine may now instill additional confidence to get vaccinated. Today’s milestone puts us one step closer to altering the course of this pandemic in the U.S.” The FDA granted this application Priority Review. The approval was granted to BioNTech Manufacturing GmbH. (August 2021)

Opdivo Approved for Adjuvant Treatment of Urothelial Carcinoma
The FDA approved Bristol-Myers Squibb’s Opdivo (nivolumab) for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection. This is the first FDA approval for adjuvant treatment of patients with high-risk UC. The results supporting this approval also supported the conversion of nivolumab’s accelerated approval for advanced/metastatic UC to a regular approval. This application was granted priority review for this indication. (August 2021)

Jemperli Gets FDA Accelerated Approval for Adults with Mismatch Repair Deficient Recurrent or Advanced Solid Tumors
The FDA grants accelerated approval to GlaxoSmithKline’s Jemperli (dostarlimab-gxly) for adults with mismatch repair deficient (dMMR) recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options.

The FDA also approved the VENTANA MMR RxDx Panel as a companion diagnostic device to select persons with dMMR solid tumors for treatment with dostarlimab-gxly.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). This application was granted priority review. (August 2021)

FDA Approves Welireg for Cancers Associated with von Hippel-Lindau Disease
Merck’s Welireg (belzutifan), a hypoxia-inducible factor inhibitor for adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery has been approved by the FDA. This application was granted priority review for this indication. (August 2021)

Lenvima Plus Keytruda Receive FDA approval for Advanced Renal Cell Carcinoma
The FDA approved the combination of Eisai’s Lenvima (lenvatinib) plus Merck’s Keytruda (pembrolizumab) for first-line treatment of adult patients with advanced renal cell carcinoma (RCC). This application was granted priority review and breakthrough therapy designation for this indication. (August 2021)

FDA Approves Keytruda for High-Risk Early-Stage Triple-Negative Breast Cancer
The FDA approved Merck’s Keytruda (pembrolizumab) for high-risk, early-stage, triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.

FDA also granted regular approval to pembrolizumab in combination with chemotherapy for people with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) as determined by an FDA approved test. FDA granted accelerated approval to pembrolizumab for this indication in November 2020.

This application was granted priority review. Pembrolizumab received breakthrough therapy designation for this indication. (July 2021)

Keytruda and Lenvima Gets FDA Regular Approval for Advanced Endometrial Carcinoma
The FDA approved Merck’s Keytruda (pembrolizumab) in combination with Eisai’s Lenvima (lenvatinib) for people with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.

FDA granted accelerated approval on September 17, 2019 to pembrolizumab with lenvatinib for advanced endometrial carcinoma. Study 309/KEYNOTE-775 (NCT03517449) was a multicenter, open-label, randomized, active-controlled trial required to confirm the clinical benefit of this accelerated approval.

This application was granted priority review. The combination of pembrolizumab and lenvatinib received breakthrough therapy designation for this indication. (July 2021)

FDA Approves Rezurock for Chronic Graft-Versus-Host Disease
The FDA approved Kadmon Pharmaceuticals’ Rezurock (belumosudil), a kinase inhibitor, for adults and people 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Australia’s Therapeutic Goods Administration, Health Canada, Switzerland’s Swissmedic, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency. This application was granted priority review and breakthrough therapy designation. (July 2021)

Darzalex Faspro Gains FDA Approval with Pomalidomide and Dexamethasone for Multiple Myeloma
The FDA approved Janssen Biotech’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) in combination with pomalidomide and dexamethasone for adults with multiple myeloma who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor. (July 2021)

Padcev Gets FDA Regular Approval for Locally Advanced or Metastatic Urothelial Cancer
The FDA approved Astellas Pharma’s Padcev (enfortumab vedotin-ejfv), a Nectin-4-directed antibody and microtubule inhibitor conjugate, for adults with locally advanced or metastatic urothelial cancer who

–– have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand (PD-L1) inhibitor and platinum-containing chemotherapy, or

–– are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy.

FDA granted accelerated approval in December 2019 to enfortumab vedotin-ejfv for people with locally advanced or metastatic urothelial cancer who have received a PD-1 or PD-L1 inhibitor and a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting.This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Health Canada and Australia’s Therapeutic Goods Administration.This application was granted priority review and breakthrough therapy designation. (July 2021)

FDA Approves Component of Treatment Regimen for Most Common Childhood Cancer
The FDA approved Jazz Pharmaceuticals’ Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) as a component of a chemotherapy regimen to treat acute lymphoblastic leukemia and lymphoblastic lymphoma in adults and children who are allergic to the E. coli-derived asparaginase products used most commonly for treatment. The only other FDA-approved drug for such patients with allergic reactions has been in global shortage for years.

“It is extremely disconcerting to patients, families and providers when there is a lack of access to critical drugs for treatment of a life-threatening, but often curable cancer, due to supply issues,” said Gregory Reaman, MD, associate director for pediatric oncology in the FDA’s Oncology Center of Excellence. “Today’s approval may provide a consistently sourced alternative to a pivotal component of potentially curative therapy for children and adults with this type of leukemia.”

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Health Canada, where the application review is pending. Rylaze received Fast Track and Orphan Drug designations for this indication. (June 2021)

Ayvakit Receives Approval for Advanced Systemic Mastocytosis
The FDA approved Blueprint Medicine’s Ayvakit™ (avapritinib) for adult people with advanced systemic mastocytosis (AdvSM), including people with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).

Avapritinib is not recommended for the treatment of patients with AdvSM with platelet counts of less than 50 X 109/L.

The recommended avapritinib dose is 200 mg orally once daily for patients with AdvSM.This application was granted priority review, breakthrough designation and orphan drug designation. (June 2021)

Truseltiq Gets Accelerated Approval for Metastatic Cholangiocarcinoma
QED Therapeutics’ Truseltiq (infigratinib) receives FDA accelerated approval, a kinase inhibitor for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

The FDA also approved Foundation Medicine’s FoundationOne® CDx for selection of people with FGFR2 fusion or other rearrangement as a companion diagnostic device for treatment with infigratinib.

This indication is approved under accelerated approval based on the overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA) and Health Canada. The application reviews are ongoing at the other regulatory agencies.

This application was granted priority review, fast-track designation, and orphan drug designation. (May 2021)

FDA Approves First Targeted Therapy for Lung Cancer Mutation Previously Considered Resistant to Drug Therapy
The FDA approved Amgen’s Lumakras (sotorasib) as the first treatment for adults with non-small cell lung cancer whose tumors have a specific type of genetic mutation called KRAS G12C and who have received at least one prior systemic therapy. This is the first approved targeted therapy for tumors with any KRAS mutation, which accounts for approximately 25% of mutations in non-small cell lung cancers. KRAS G12C mutations represent about 13% of mutations in non-small cell lung cancers.

“KRAS mutations have long been considered resistant to drug therapy, representing a true unmet need for patients with certain types of cancer,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Today’s approval represents a significant step towards a future where more patients will have a personalized treatment approach.”

Lung cancer, the most common cancer type with the highest mortality, can largely be categorized by the genetic mutations that cause it. KRAS is a type of mutation in a group of genes that help regulate cell growth and division.

As part of the evaluation for this accelerated approval, the agency is requiring a postmarketing trial to investigate whether a lower dose will have a similar clinical effect.

Lumakras was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is unmet medical need and a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients. Further study is required to verify and describe anticipated clinical benefits of Lumakras.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations. Lumakras also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada and Medicines and Healthcare products Regulatory Agency (MHRA; United Kingdom). The application reviews are ongoing at the other regulatory agencies.

Along with Lumakras, the FDA also approved the QIAGEN therascreen KRAS RGQ PCR kit and Guardant Health’s Guardant360 CDx as companion diagnostics for Lumakras today. The QIAGEN GmbH test analyzes tumor tissue and the Guardant Health, Inc. test analyzes plasma specimens to determine if Lumakras is an appropriate treatment for patients. If no mutation is detected in a plasma specimen, the patient’s tumor should be tested. (May 2021)

First Targeted Therapy for Subset of Non-Small Cell Lung Cancer Gains FDA Approval
The FDA approved Janssen’s Rybrevant (amivantamab-vmjw) as the first treatment for adults with non-small cell lung cancer whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

The FDA also approved the Guardant Health Guardant360 CDx as a companion diagnostic for Rybrevant today.

“With today’s approval, for the first time, patients with non-small cell lung cancer with EGFR exon 20 insertion mutations will have a targeted treatment option,” said Julia Beaver, MD, chief of medical oncology in the FDA’s Oncology Center of Excellence and acting deputy director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

Lung cancer is the most common cancer type and the leading cause of cancer-related deaths worldwide, with non-small cell lung cancer accounting for 80% to 85% of all lung cancers, according to the American Cancer Society. Approximately 2% to 3% of patients with non-small cell lung cancer will have EGFR exon 20 insertion mutations, which are a group of mutations on a protein that causes rapid cell growth, and consequently, helps cancer spread. EGFR exon 20 insertion mutations are the third most common type of EGFR mutation.

The FDA granted approval of Rybrevant to Janssen Pharmaceutical Companies of Johnson & Johnson.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, the FDA collaborated with the Brazilian Health Regulatory Agency and United Kingdom’s Medicines and Healthcare products Regulatory Agency. The application reviews are ongoing at the other regulatory agencies. (May 2021)

Opdivo Approved for Resected Esophageal or GEJ Cancer
Bristol Myers Squibb’s Opdivo (nivolumab) for people with completely resected esophageal or gastroesophageal junction (GEJ) cancer with residual pathologic disease who have received neoadjuvant chemoradiotherapy.

The recommended nivolumab dose for adjuvant treatment of resected esophageal or GEJ cancer is 240 mg every 2 weeks or 480 mg every 4 weeks for a total treatment duration of 1 year. Both doses are administered as 30-minute intravenous infusions.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. (May 2021)

FDA Grants Accelerated Approval to Keytruda for HER2-positive Gastric Cancer
The FDA has granted accelerated approval to Merck’s Keytruda (pembrolizumab) in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.

The recommended pembrolizumab dose for adult patients with locally advanced unresectable or metastatic HER2 positive gastric or GEJ adenocarcinoma in combination with trastuzumab and chemotherapy is 200 mg every 3 weeks or 400 mg every 6 weeks. This application was granted priority review. (May 2021)

Zynlonta Gains Accelerated Approval for Large B-cell Lymphoma
ADC Therapeutics was granted approval for Zynlonta (loncastuximab tesirine-lpyl), a CD19-directed antibody and alkylating agent conjugate, for adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from low grade lymphoma, and high-grade B-cell lymphoma.

This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). This application was granted priority review and orphan drug designation. (Apr. 2021)

FDA Approves Immunotherapy for Endometrial Cancer with Specific Biomarker
The FDA granted accelerated approval to GlaxoSmithKline’s Jemperli (dostarlimab) for treating people with recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum-containing chemotherapy and whose cancers have a specific genetic feature known as dMMR (which contain abnormalities that affect the proper repair of DNA inside the cell), as determined by an FDA-approved test.

“Today’s approval of Jemperli is evidence of the FDA’s progress in applying precision medicine to expand treatment options for patients with cancer,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “This immunotherapy was specifically studied to target dMMR endometrial cancer and leverages scientific knowledge surrounding the mechanism of immunotherapy response in this unmet medical need population.”

Endometrial cancer is the most common gynecologic malignancy in the United States and its prevalence is increasing. Approximately 75% of endometrial cancers are diagnosed at an early stage and are typically curable with surgery. However, women with advanced and recurrent endometrial cancer have limited therapeutic options following front-line standard treatment with a platinum-containing chemotherapeutic regimen. Approximately 25% to 30% of patients with advanced endometrial cancer have dMMR tumors.

Jemperli works by targeting the cellular pathway known as PD-1/PD-L1 (proteins found on the body’s immune cells and some cancer cells). Jemperli helps the body’s immune system in its fight against cancer cells by blocking this pathway.

Jemperli received Priority Review designation and Breakthrough Therapy designation for this indication. (Apr. 2021)

FDA Approves First Immunotherapy for Initial Treatment of Gastric Cancer
Bristol Myers Squibb’s Opdivo (nivolumab) was FDA approved, in combination with certain types of chemotherapy, for the initial treatment of people with advanced or metastatic gastric cancer, gastroesophageal junction cancer and esophageal adenocarcinoma. This is the first FDA-approved immunotherapy for the first-line treatment of gastric cancer.

“Today’s approval is the first treatment in more than a decade to show a survival benefit for patients with advanced or metastatic gastric cancer who are being treated for the first time,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

Opdivo received Priority Review and Orphan Drug designations for this indication. Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, the FDA collaborated with the Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency, Health Canada and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. (Apr. 2021)

Trodelvy Granted Accelerated Approval for Advanced Urothelial Cancer
The FDA granted accelerated approval to Immunomedics’s Trodelvy (sacituzumab govitecan) for people with locally advanced or metastatic urothelial cancer (mUC) who previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. This application was granted priority review and fast track designation. (Apr. 2021)

FDA Grants Regular Approval to Trodelvy for Triple-Negative Breast Cancer
Immunomedic’s Trodelvy (sacituzumab govitecan) received regular FDA approval for people with unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease.

In April 2020, sacituzumab govitecan received accelerated approval for people with mTNBC who have received at least two prior therapies for metastatic disease. The following trial was the confirmatory trial for the accelerated approval. This application was granted priority review and breakthrough designation. Apr. 2021)

FDA Approves New Dosing Regimen for Erbitux
The FDA approved a new dosage regimen of 500 mg/m2 as a 120-minute intravenous infusion every two weeks (Q2W) for ImClone’s Erbitux (cetuximab) for people with K-Ras wild-type, EGFR-expressing colorectal cancer (mCRC) or squamous cell carcinoma of the head and neck (SCCHN).

This approval provides for a biweekly dosage regimen option in addition to the previously approved weekly dosage regimen for the approved indications when cetuximab is used as a single agent or in combination with chemotherapy. (Apr. 2021)

Sarclisa Approved for Multiple Myeloma
The FDA approved Sanofi-Aventis’ Sarclisa (isatuximab-irfc), in combination with carfilzomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy.

The recommended isatuximab-irfc dose with carfilzomib and dexamethasone is 10 mg/kg as an intravenous infusion every week for 4 weeks followed by every 2 weeks until disease progression or unacceptable toxicity. Sarclisa has orphan drug designation for the treatment of multiple myeloma. (Mar. 2021)

FDA Approves First Cell-Based Gene Therapy for Adults with Multiple Myeloma
The FDA granted approval of Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adults with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy, to Celgene Corporation, a Bristol Myers Squibb company. Abecma is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma.

“While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual’s age and the stage of the condition at the time of diagnosis. Today’s approval provides a new treatment option for patients who have this uncommon type of cancer,” says Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research.

The FDA is requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Abecma. Abecma was granted Orphan Drug and Breakthrough Therapy designations by the FDA. Orphan Drug designation provides incentives to assist and encourage the development of drugs for rare diseases. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Breakthrough Therapy designation was granted based on sustained responses observed in patients with relapsed and refractory myeloma. (Mar. 2021)

Keytruda Approved for Esophageal or GEJ Carcinoma
The FDA has approved Merck’s Keytruda (pembrolizumab) in combination with platinum and fluoropyrimidine-based chemotherapy for people with metastatic or locally advanced esophageal or gastroesophageal (GEJ) (tumors with epicenter 1 to 5 centimeters above the gastroesophageal junction) carcinoma who are not candidates for surgical resection or definitive chemoradiation.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and Switzerland’s Swissmedic. The application reviews are ongoing at the other regulatory agencies. This application was granted priority review. (Mar. 2021)

FDA Approves Lorbrena for Metastatic ALK-Positive Non-Small Cell Lung Cancer
The FDA has granted regular approval to Pfizer’s Lorbrena (lorlatinib) for the treatment of people with metastatic non-small cell lung cancer whose tumors are anaplastic lymphoma kinase (ALK)-positive, as detected by an FDA-approved test. The FDA also approved Ventana Medical Systems’ Ventana ALK (D5F3) CDx Assay as a companion diagnostic for Lorbrena.

Lorbrena previously received accelerated approval in November 2018 for the second- or third-line treatment of ALK-positive metastatic non-small cell lung cancer. This expanded approval of Lorbrena is based on the results from a Phase III trial, which showed a 72-percent reduction in risk of progression or death versus crizotinib in a previously untreated patient population. (Mar. 2021)

FDA Grants Accelerated Approval to Yescarta for Relapsed or Refractory Follicular Lymphoma
Yescarta (axicabtagene ciloleucel) has gained FDA approval to treat adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Yescarta is a CD19-directed chimeric antigen receptor (CAR) T-cell therapy manufactured by Kite Pharma. This accelerated approval was based on a single-arm, open-label, multicenter trial in which 91 percent of people with relapsed or refractory follicular lymphoma responded to Yescarta. (Mar. 2021)

Fotivda Approved to Treat Relapsed or Refractory Advanced Renal Cell Carcinoma
The FDA has approved AVEO Pharmaceuticals’ Fotivda (tivozanib), a kinase inhibitor, for adults with relapsed or refractory advanced renal cell carcinoma following two or more prior systemic therapies.

The approval of Fotivda is based on a Phase III study comparing Fotivda to sorafenib in relapsed or refractory advanced renal cell carcinoma following two or more prior systemic therapies. The main efficacy outcome measure was progression-free survival. Median progression-free survival was 5.6 months in the Fotivda arm, compared with 3.9 months for those treated with sorafenib. Median overall survival was 16.4 and 19.2 months for the Fotivda and sorafenib arms, respectively. The objective response rate was 18 percent for the Fotivda arm and 8 percent for the sorafenib arm. (Mar. 2021)

FDA Grants Accelerated Approval to Tepmetko for Metastatic Non-Small Cell Lung Cancer
EMD Serono’s Tepmetko (tepotinib) has received FDA approval to treat adults with metastatic non-small cell lung cancer harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. (Feb. 2021)

Ukoniq Granted Accelerated Approval to Treat Marginal Zone Lymphoma and Follicular Lymphoma
The FDA has granted accelerated approval to TG Therapeutics’ Ukoniq (umbralisib), a kinase inhibitor including PI3K-delta and casein kinase CK1-epsilon, for the following indications:

• Adults with relapsed or refractory marginal zone lymphoma who have received at least one prior anti-CD20-based regimen
• Adults with relapsed or refractory follicular lymphoma who have received at least three prior lines of systemic therapy

Accelerated approval was granted for these indications based on overall response rate data from a Phase II trial. Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial. (Feb. 2021)

Libtayo Approved to Treat Locally Advanced and Metastatic Basal Cell Carcinoma
Regeneron Pharmaceuticals’ Libtayo (cemiplimab-rwlc) has been approved by the FDA to treat people with locally advanced basal cell carcinoma previously treated with a hedgehog pathway inhibitor, or for whom a hedgehog pathway inhibitor is not appropriate. The FDA also granted Libtayo accelerated approval to treat people with metastatic basal cell carcinoma previously treated with a hedgehog pathway inhibitor, or for whom a hedgehog pathway inhibitor is not appropriate. (Feb. 2021)

FDA Approves New Drug to Reduce Bone Marrow Suppression Caused by Chemotherapy
The FDA has approved Cosela (trilaciclib) as the first therapy in its class to reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage small cell lung cancer. Cosela, which is manufactured by G1 Therapeutics, may help protect bone marrow cells from damage caused by chemotherapy by inhibiting cyclin-dependent kinase 4/6, a type of enzyme.

“For patients with extensive-stage small cell lung cancer, protecting bone marrow function may help make their chemotherapy safer and allow them to complete their course of treatment on time and according to plan,” says Albert Deisseroth, MD, PhD, supervisory medical officer in the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.

The effectiveness of Cosela was evaluated in three randomized, double-blind, placebo-controlled studies in people with extensive-stage small cell lung cancer. Combined, these studies randomly assigned 245 people to receive either an infusion of Cosela in their veins or a placebo before chemotherapy. The studies then compared the two groups for the proportion of patients with severe neutropenia and the duration of severe neutropenia in the first cycle of chemotherapy. In all three studies, trial participants who received Cosela had a lower chance of having severe neutropenia compared to those who received a placebo. Among those who had severe neutropenia, people who received Cosela, on average, had it for a shorter time than those who received a placebo. (Feb. 2021)

FDA Approves Libtayo for Non-Small Cell Lung Cancer with High PD-L1 Expression
Regeneron Pharmaceuticals’ PD-1 inhibitor Libtayo (cemiplimab-rwlc) has received FDA approval as a first-line treatment of people with locally advanced non-small cell lung cancer whose tumors have high PD-L1 expression, as determined by an FDA-approved test. Eligible non-small cell lung cancer survivors must either have metastatic or locally advanced tumors that are not candidates for surgical resection or definitive chemoradiation, and the tumors must not have EGFR, ALK, or ROS1 aberrations.

This approval was based on an analysis of 710 people with locally advanced non-small cell lung cancer who were not candidates for surgical resection or definitive chemoradiation, or with metastatic non-small cell lung cancer, who were randomized to receive treatment in a Phase III trial. Eligible participants were intended to have PD-L1 expression of ≥50%. In this population, Libtayo reduced the risk of death by 32 percent compared to chemotherapy. (Feb. 2021)

Pepaxto Combination Receives Accelerated Approval for Relapsed or Refractory Multiple Myeloma
The FDA has granted accelerated approval to Oncopeptides’ Pepaxto (melphalan flufenamide), in combination with dexamethasone, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody.

This indication has been granted under accelerated approval based upon a clinical trial evaluating intravenous Pepaxto in combination with dexamethasone, which included heavily pre-treated patients with a poor prognosis. The study looked at 157 people with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The overall response rate for those within this group of people with refractory multiple myeloma was 23.7 percent and the median duration of response was 4.2 months.

Pepaxto was approved under accelerated approval, which allows for earlier approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. (Feb. 2021)

New Treatment Approved for Adults with Relapsed or Refractory Large-B-Cell Lymphoma
The U.S. Food and Drug Administration has approved Juno Therapeutics’ Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy, to treat adults with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other types of systemic treatment. Breyanzi is a chimeric antigen receptor (CAR) T-cell therapy that uses a person’s own T-cells to help fight the lymphoma.

The safety and efficacy of Breyanzi were established in a multicenter clinical trial of more than 250 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Breyanzi was 54 percent. Breyanzi is not indicated for the treatment of people with primary central nervous system lymphoma. (Jan. 2021)

FDA Approves Enhertu for HER2-Positive Gastric Adenocarcinomas
The FDA has approved Daiichi Sankyo’s Enhertu (fam-trastuzumab deruxtecan-nxki) for adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal adenocarcinoma who have received a prior trastuzumab-based regimen. Approval was based on the positive results of a Phase II trial in which Enhertu demonstrated a statistically significant and clinically meaningful improvement in both overall survival and objective response rate versus chemotherapy (irinotecan or paclitaxel) in people with advanced gastric cancer or gastroesophageal adenocarcinoma who had progressed on at least two or more prior regimens, including trastuzumab plus a fluoropyrimidine- and platinum-based chemotherapy combination. (Jan. 2021)

Opdivo Plus Cabometyx Combination Gains FDA Approval for the Treatment of Advanced Renal Cell Carcinoma
The combination of Bristol-Myers Squibb’s Opdivo (nivolumab) and Exelixis’s Cabometyx (cabozantinib) has been cleared by the FDA as a first-line treatment for people with advanced renal cell carcinoma. Approval was based on Phase III trial results which demonstrated that the Opdivo plus Cabometyx combination doubled median progression-free survival and objective response rate and showed superior overall survival versus sunitinib in people with previously untreated advanced renal cell carcinoma. (Jan. 2021)

FDA Approves Xalkori for Children and Young Adults with Relapsed or Refractory, Systemic Anaplastic Large Cell Lymphoma
The FDA has approved Pfizer’s Xalkori (crizotinib) for children, one year of age and older, and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma that is ALK-positive. The safety and efficacy of Xalkori have not been established in older adults with relapsed or refractory, systemic ALK-positive anaplastic large cell lymphoma.

The FDA approval is based on results from a multicenter study of 121 participants between the ages of 1 and 21, which included 26 people with relapsed or refractory, systemic ALK-positive anaplastic large cell lymphoma after at least one systemic treatment. Treatment with Xalkori resulted in an objective response rate of 88 percent. Among the 23 study participants who achieved a response, 39 percent maintained their response for at least 6 months, and 22 percent maintained their response for at least 12 months. (Jan. 2021)

Darzalex Faspro Granted Accelerated Approval to Treat Newly Diagnosed Light Chain Amyloidosis
The FDA has granted accelerated approval to Janssen Biotech’s Darzalex Faspro (daratumumab plus hyaluronidase) in combination with bortezomib, cyclophosphamide, and dexamethasone for newly diagnosed light chain amyloidosis. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

This approval is based on positive results from a Phase III study that evaluated Darzalex Faspro in combination with bortezomib, cyclophosphamide, and dexamethasone, compared with bortezomib, cyclophosphamide, and dexamethasone alone, a common treatment regimen used in adults with newly diagnosed light chain amyloidosis. Trial participants receiving treatment with Darzalex Faspro experienced a hematologic complete response rate more than triple that of those receiving bortezomib, cyclophosphamide, and dexamethasone alone. (Jan. 2021)q