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Osimertinib Plus Platinum-Based Chemotherapy Approved for Locally Advanced or Metastatic Non–Small Cell Lung Cancer
Lyn Brook
FDA Updates
The FDA has granted approval to osimertinib (Tagrisso®, AstraZeneca Pharmaceuticals LP) with platinum-based chemotherapy for patients with locally advanced or metastatic non–small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test. &nbs...
Lifileucel Granted Accelerated Approval for Unresectable or Metastatic Melanoma
Lyn Brook
FDA Updates
The FDA has granted accelerated approval to lifileucel (Amtagvi, Iovance Biotherapeutics, Inc.) for adult patients with unresectable or metastatic melanoma previously treated with a programmed cell death protein 1 (PD-1) blocking antibody, and if BRAF V600–positive, a BRAF inhibitor with or without a mitogen-activated protein kinase (MEK) inhibitor...
Tepotinib Granted Traditional Approval for Metastatic Non–Small Cell Lung Cancer
Lyn Brook
FDA Updates
The FDA has granted traditional approval to tepotinib (Tepmetko®, EMD Serono, Inc.) for adult patients 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.   Why it matters...
Irinotecan Liposome Approved for First-Line Metastatic Pancreatic Adenocarcinoma Treatment
Lyn Brook
FDA Updates
The FDA has approved irinotecan liposome (Onivyde®, Ipsen Biopharmaceuticals, Inc.) with oxaliplatin, fluorouracil, and leucovorin, for the first-line treatment of metastatic pancreatic adenocarcinoma.   Why it matters: "The approval of liposomal irinotecan in combination with standard-of-care agents provides clinicians and patients with anoth...
Erdafitinib Approved for Locally Advanced or Metastatic Urothelial Cancer
Lyn Brook
FDA Updates
The FDA has granted approval to erdafitinib (Balversa™, Janssen Biotech) for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one...
Pembrolizumab Plus Chemoradiotherapy Approved for FIGO 2014 Stage III-IVA Cervical Cancer
Lyn Brook
FDA Updates
The FDA has granted approval for pembrolizumab (Keytruda®, Merck) with chemoradiotherapy (CRT) for patients with International Federation of Gynecology and Obstetrics (FIGO) 2014 Stage III-IVA cervical cancer. For background: "Pembrolizumab has shown efficacy in patients with cervical cancer," said Domenica Lorusso, MD, PhD, an Associate Professor ...
Enfortumab Vedotin-ejfv Plus Pembrolizumab Approved for Urothelial Cancer
Lyn Brook
FDA Updates
The FDA has approved enfortumab vedotin-ejfv (Padcev®, Astellas Pharma) in combination with pembrolizumab (Keytruda®, Merck) for patients with locally advanced or metastatic urothelial cancer (UC). The FDA previously granted accelerated approval for this therapy combination for patients with locally advanced or metastatic UC who are ineligible for ...
Belzutifan Approved for Advanced Renal Cell Carcinoma
Lyn Brook
FDA Updates
The FDA has approved belzutifan (Welireg™, Merck & Co., Inc.) for advanced renal cell carcinoma (RCC) subsequent to 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).   Why it matters: "Belzutifan is a first-in-class oral hypoxia...
Eflornithine Approved for Reduction of Risk of Relapse in High-Risk Neuroblastoma
Lyn Brook
FDA Updates
The FDA has granted approval to eflornithine (Iwilfin™, US World Meds, LLC) for reduction of 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 marks the first approval of a therapy...
Pirtobrutinib Granted Accelerated Approval for Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma
Lyn Brook
FDA Updates
The FDA has granted accelerated approval to pirtobrutinib (Jaypirca®, Eli Lilly and Company) for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor.   Why it matters...
Nirogacestat Approved for Desmoid Tumors
Lyn Brook
FDA Updates
The FDA has granted approval to nirogacestat (Ogsiveo™, SpringWorks Therapeutics, Inc.) for adult patients with progressing desmoid tumors who require systemic treatment. This is the first approval for nirogacestat and the first approved treatment for desmoid tumors.   Why it matters: "Management of desmoid tumors is challenging because of the...
Enzalutamide Approved for Non-Metastatic Castration-Sensitive Prostate Cancer with Biochemical Recurrence
Lyn Brook
FDA Updates
The FDA has approved enzalutamide (Xtandi®, Astellas Pharma US, Inc.) for nonmetastatic castration-sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastasis (high-risk BCR).   Why it matters: "Within 10 years following definitive therapy for prostate cancer, 20% to 50% of patients have BCR characterized by a ri...
Capivasertib Plus Fulvestrant Approved for Breast Cancer
Lyn Brook
FDA Updates
The FDA has approved capivasertib (Truqap™, AstraZeneca Pharmaceuticals) in combination with fulvestrant for adult patients with hormone receptor (HR)–positive, human epidermal growth factor receptor 2 (HER2)–negative locally advanced or metastatic breast cancer. This indication is for patients with one or more PIK3CA/AKT1/PTEN alterations, as dete...
Pembrolizumab Approved for HER2-Negative Gastric and Gastroesophageal Junction Cancer
Lyn Brook
FDA Updates
Pembrolizumab. The FDA has approved pembrolizumab (Keytruda®, Merck) with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor 2 (HER2)–negative gastric or gastroesophageal junction (GEJ) adenocarcinoma.Why it matters: "Programmed cell death protein 1 (PD-1) inhibitors combined with chemotherapy have shown efficacy in gastric or gastroesophageal junction cancer," wrote Sun Young Rha, MD, the Director of Songdang Institute for Cancer Research at the Yonsei University College of Medicine, Yonsei University Health System in Seoul, Korea, and colleagues, in their published results of the KEYNOTE-859 trial (NCT03675737), on which approval was based. "We compared the efficacy and safety of pembrolizumab plus chemotherapy with placebo plus chemotherapy in participants with locally advanced or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma."What they studied: Efficacy was studied in the phase 3, multicenter, double-blind, placebo-controlled trial in which 1,579 patients with HER2-negative advanced gastric or GEJ adenocarcinoma who had not previously received systemic therapy for metastatic disease were enrolled. Patients were randomized 1:1 to receive either 200 mg of pembrolizumab or placebo in addition to investigator's choice of combination chemotherapy consisting of either 80 mg/m2 of cisplatin plus 5-fluorouracil (5-FU) 800 mg/m2/day for five days, or 130 mg/m2 of oxaliplatin on Day 1 plus 1,000 mg/m2 of capecitabine twice a day for 14 days of each 21-day cycle.The primary end point measured was overall survival. Secondary end points included progression-free survival, overall response rate, and duration of response as per RECIST v1.1, modified to follow a maximum of 10 target lesions and a maximum of five target lesions per organ, as assessed by blinded independent central review.What they found: Those being treated with pembrolizumab saw statistically significant results in progression-free survival, overall survival, and overall response. The median overall survival was 12.9 months with pembrolizumab compared with 11.5 months in those being treated with placebo. The median progression-free survival was 6.9 months in those being treated with pembrolizumab compared with 5.6 months in those being treated with placebo. Overall response rate was seen at 51% in the pembrolizumab arm and 42% in the placebo arm. The median duration of response in the pembrolizumab arm was 8 months compared with 5.7 months in the placebo arm.Additionally, in a prespecified analysis, a statistically significant improvement was observed in overall survival, progression-free survival, and overall response rate in patients receiving pembrolizumab based on tumors expressing programmed death-ligand 1 (PD-L1) combined positive score (CPS) ≥1 and CPS ≥10.Adverse events: Pembrolizumab was permanently discontinued in 15% of patients due to adverse events. Permanent discontinuation due to adverse events in ≥1% of patients were infections and diarrhea. Conclusion: "Participants in the pembrolizumab plus chemotherapy group had a significant and clinically meaningful improvement in overall survival with manageable toxicity compared with participants in the placebo plus chemotherapy group," concluded Dr. Rha and colleagues. "Therefore, pembrolizumab with chemotherapy might be a first-line treatment option for patients with locally advanced or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma."Instructions: The recommended dosage of pembrolizumab is 200 mg every three weeks or 400 mg every six weeks until disease progression or unacceptable toxicity. When given on the same day, pembrolizumab should be administered prior to chemotherapy.For More InformationRha SY, Oh DY, Yanez P, et al (2023). Pembrolizumab plus chemotherapy versus placebo plus chemotherapy for HER2-negative advanced gastric cancer (KEYNOTE-859): a multicentre, randomised, double-blind, phase 3 trial. Lancet, 24:11, 1181-1195. DOI:10.1016/S1470-2045(23)00515-6Clinicaltrials.gov (2023). Pembrolizumab (MK-3475) plus chemotherapy versus placebo plus chemotherapy in participants gastric or gastroesophageal junction (GEJ) adenocarcinoma (MK-3475-859/KEYNOTE-859). NLM identifier: NCT03675737.Keytruda® (pembrolizumab) prescribing information (2023). Merck. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/125514s143lbl.pdfUS Food and Drug Administration (2023). FDA approves pembrolizumab with chemotherapy for HER2-negative gastric or gastroesophageal junction adenocarcinoma. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-chemotherapy-her2-negative-gastric-or-gastroesophageal-junction Image credit: Fvasconcellos. Licensed under CC BY-SA. Licensed under the public domain
Repotrectinib Approved for ROS1-Positive Non–Small Cell Lung Cancer
Lyn Brook
FDA Updates
The FDA has approved repotrectinib (Augtyro™, Bristol Myers Squibb Company) for locally advanced or metastatic ROS1-positive non–small cell lung cancer (NSCLC). This is the first approval for the next-generation ROS1/TRK tyrosine kinase inhibitor (TKI) repotrectinib and is the first FDA approval to include patients with ROS1-positive NSCLC who have...
Revision of Pembrolizumab Indication for Gastric Cancer
Lyn Brook
FDA Updates
The FDA has amended the existing indication of pembrolizumab (Keytruda®, Merck) with trastuzumab, fluoropyrimidine, and platinum-containing chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)–positive gastric or gastroesophageal junction (GEJ) adenoca...
Fruquintinib Approved for Refractory Metastatic Colorectal Cancer
Lyn Brook
FDA Updates
The FDA has approved fruquintinib (Fruzaqla™, Takeda Pharmaceuticals, Inc.) for adult patients with metastatic colorectal cancer (mCRC) who received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti–vascular endothelial growth factor (VEGF) therapy, and, if rat sarcoma (RAS) wild-type and medically appropriate, an an...
Pembrolizumab Approved for Biliary Tract Cancer
Lyn Brook
FDA Updates
The FDA has approved pembrolizumab (Keytruda®, Merck) in combination with gemcitabine and cisplatin for locally advanced unresectable or metastatic biliary tract cancer (BTC).   Why it matters: "Biliary tract cancers, which arise from the intrahepatic or extrahepatic bile ducts and the gallbladder, generally have a poor prognosis and are risin...
Toripalimab-tpzi Approved for Nasopharyngeal Carcinoma
Lyn Brook
FDA Updates
The FDA has approved toripalimab-tpzi (Loqtorzi™, Coherus BioSciences, Inc.) in combination with cisplatin and gemcitabine for first-line treatment of metastatic or recurrent, locally advanced nasopharyngeal carcinoma (NPC). Additionally, the FDA approved toripalimab-tpzi as a single agent for treatment of recurrent unresectable or metastatic NPC w...
Ivosidenib Approved for IDH1-Mutant Relapsed or Refractory Myelodysplastic Syndromes
Lyn Brook
FDA Updates
The FDA has approved ivosidenib (Tibsovo®, Servier Pharmaceuticals LLC) for adult patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, as detected by an FDA-approved test. In addition, the FDA approved a companion diagnostic device, Abbott RealTime IDH1 Assay, to select ...