Here’s a look back on the FDA happenings from the month of May 2020.
The month of May 2020 was an exciting month for oncology with many updates from the FDA. In particular, the FDA granted 4 approvals on Friday, May 15, 2020 alone. Eight additional approvals were granted throughout the month. Among other FDA news this month, the FDA granted a Priority Review, 2 Orphan Drug designations, a Breakthrough Therapy designation, a Fast Track designation, a Regenerative Medicine Advanced Therapy designation, and more.
On May 1, 2020, the FDA granted approval to an electrical impedance spectroscopy system, Nevisense 3.0, the third-generation of the SciBase Nevisense system, for the early detection of melanoma. Nevisense uses an AI-based point-of-care system for a non-invasive procedure to evaluate irregular moles and is the only FDA-approved system available in the United States for the detection of melanoma.
The FDA granted Priority Review on May 1, 2020, to an investigational oral hypomethylating agent, CC-486, as a maintenance treatment for adult patients with acute myeloid leukemia who achieved a complete remission (CR) or CR with incomplete blood count recovery after receiving induction therapy with or without consolidation therapy.
On May 4, 2020, the FDA granted an Orphan Drug designation to a third-generation BCR-ABL inhibitor, HQP1351, as treatment of patients with chronic myeloid leukemia.
The action date for the CD19-directed chimeric antigen receptor (CAR) T cell therapy lisocabtagene maraleucel (liso-cel) as treatment of adult patients with relapsed or refractory large B-cell lymphoma after at least 2 therapies, was extended 3 months by the FDA. The updated Prescription Drug User Fee Act (PDUFA) action date is November 16, 2020, according to an announcement on May 6, 2020.
On May 6, 2020, MIV-818, was granted an Orphan Drug Designation by the FDA for the treatment of patients with hepatocellular carcinoma, which follows an Orphan Medicinal Drug designation in Europe for the same indication, which was granted in April 2020 by the Committee for Orphan Medicinal Products, a division of the European Medicines Agency.
The FDA granted approval on May 6, 2020, to capmatinib (Tabrecta) for adult patients with metastatic non–small cell lung cancer (NSCLC) whose tumors have a mutation that leads to MET exon 14 skipping, as detected by an FDA-approved test.
On May 8, 2020, the FDA granted a Regenerative Medicine Advanced Therapy designation to ilixadencel, as treatment of metastatic renal cell carcinoma.
The FDA granted approval on May 8, 2020, to the combination of olaparib (Lynparza) and bevacizumab (Avastin) for the maintenance treatment of patients with advanced ovarian cancer who are in complete or partial response to first-line platinum-based chemotherapy with bevacizumab and whose cancer is associated with homologous recombination deficiency–positive status defined by either a deleterious or suspected deleterious BRCA mutation, and/or genomic instability.
On May 9, 2020, the FDA approved selpercatinib (formerly known as LOXO-292; Retevmo) capsules for the treatment of patients with lung cancer or thyroid cancer harboring RET alterations, marking the first treatment approved by the FDA to target RET gene alterations.
The FDA granted a Breakthrough Therapy designation on May 11, 2020, to fam-trastuzumab deruxtecan-nxki (Enhertu) for the treatment of patients with HER2-positive unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma who have received 2 or more prior regimens including trastuzumab.
On May 13, 2020, the FDA issued a Refusal to File letter regarding the Biologics License Application for the CAR T-cell agent, idecabtagene vicleucel (ide-cel), which was submitted in March 2020 as a potential treatment for patients with heavily pretreated relapsed or refractory multiple myeloma. A preliminary review was carried out by the FDA, but it was realized that the Chemistry, Manufacturing, and Control module of the BLA required more information before the review could be completed.
The FDA issued a complete response letter (CRL) to Blueprint Medicines, Inc regarding the New Drug Application for avapritinib (Ayvakit) as treatment of adult patients with unresectable or metastatic fourth-line gastrointestinal stromal tumor. The CRL denies approval of the application.
On May 15, 2020, the FDA granted approval to pomalidomide (Pomalyst) for the treatment of patients with acquired immunodeficiency syndromes–related Kaposi sarcoma who have developed resistance to highly active antiretroviral therapy, or those with Kaposi sarcoma who are human immunodeficiency virus–negative.
The FDA approved the combination of nivolumab (Opdivo) and ipilimumab (Yervoy) on May 15, 2020, for the treatment of patients with metastatic or recurrent NSCLC whose tumors express PD-L1 (≥1%), as determined by an FDA-approved test, and who do not have an EGFR or ALK tumor aberration.
On May 15, 2020, the FDA has approved rucaparib (Rubraca) the treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy.
On May 15, 2020, the FDA has granted approval to ripretinib (Qinlock) for the treatment of adult patients with advanced gastrointestinal stromal tumor who had at least 3 prior lines of therapy with kinase inhibitors, including imatinib (Gleevec). The approval is 3 months ahead of the Prescription Drug Fee Act target action date.
The FDA granted approval on May 18, 2020, to atezolizumab (Tecentriq) monotherapy as treatment for adults with metastatic non–small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression (PD-L1 staining on ≥50% of tumor cells [TC ≥50%] or PD-L1 staining on tumor-infiltrating immune cells [IC] covering ≥10% of the tumor area [IC ≥10%]), as determined by an FDA-approved test, and have no EGFR or ALK genomic tumor aberrations. This approval marks the fifth indication for atezolizumab in metastatic NSCLC and lung cancer in general, Genentech announced in a press release.
The FDA granted an Orphan Drug designation to fam-trastuzumab deruxtecan-nxki (Enhertu) for the treatment of patients with gastric or gastroesophageal junction cancer on May 22, 2020.
The FDA has approved the combination of nivolumab (Opdivo) and ipilimumab (Yervoy) with limited platinum-doublet chemotherapy for the frontline treatment of patients with metastatic or recurrent NSCLC who do not have EGFR or ALK genomic tumor aberrations, according to an announcement on May 26, 2020.
On May 28, 2020, the FDA has announced that they will no longer hold an Oncologic Drugs Advisory Committee meeting to discuss the Biologics License Application for margetuximab (MGAH22) in combination with chemotherapy as a potential treatment of patients with HER2-positive breast cancer. However, the FDA still plans to meet the Prescription Drug User Fee Act date of December 18, 2020, for the application review.
The FDA granted a Fast Track designation to onvansertib for the second-line treatment of patients with KRAS-mutant metastatic colorectal cancer in combination with FOLFIRI (5-fluorouracil, leucovorin, and irinotecan) and bevacizumab (Avastin) on May 28, 2020.
The FDA granted approval to atezolizumab in combination with bevacizumab as treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not had a prior systemic therapyon May 29, 2020.
Pralsetinib (BLU-667) was granted a priority review on May 29, 2020, for the treatment of patients with advanced solid tumors harboring RET alterations, with a planned action date of November 23, 2020.
The FDA approved ramucirumab (Cyramza) in combination with erlotinib (Tarceva) for the first-line treatment of patients with metastatic NSCLC harboring EGFR exon 19 deletions or exon 21 (L858R) mutations on May 30, 2020, marking this the only anti–VEGFR/EGFR tyrosine kinase inhibitor combination regimen approved by the FDA for the treatment of patients with EGFR-mutant metastatic NSCLC.