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.
FDA Approves EIS System Nevisense 3.0 for the Early Detection of Melanoma
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.
FDA Grants Priority Review to Oral Hypomethylating Agent for Frontline Maintenance in AML
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.
FDA Grants Orphan Drug Designation to HQP1351 in Chronic Myeloid Leukemia
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.
FDA Extends Target Action Date for Liso-cel in R/R Large B-cell Lymphoma
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.
FDA Grants Orphan Drug Designation to First Liver-Targeted Drug for HCC
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.
FDA Approves Capmatinib in MET Exon 14+ Metastatic Non-Small Cell Lung Cancer
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.
FDA Grants RMAT Designation to Ilixadencel in Metastatic RCC
On May 8, 2020, the FDA granted a Regenerative Medicine Advanced Therapy designation to ilixadencel, as treatment of metastatic renal cell carcinoma.
FDA Approves Maintenance Olaparib/Bevacizumab in Advanced Ovarian Cancer
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.
FDA Approves Selpercatinib for RET+ Lung and Thyroid Cancers
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.
FDA Grants Breakthrough Therapy Designation to Trastuzumab Deruxtecan in HER2+ Gastric Cancer
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.
FDA Halts Review of BLA for Ide-cel in R/R Multiple Myeloma Pending Additional Data
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.
FDA Denies Approval of Avapritinib in Fourth-Line GIST
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.
FDA Approves Pomalidomide for Treatment of Kaposi Sarcoma
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.
FDA Approves Nivolumab/Ipilimumab Combo for Advanced PD-L1+ NSCLC
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.
FDA Approves Rucaparib for BRCA1/2-Mutant mCRPC
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.
Ripretinib Receives FDA Approval for Treatment of Advanced GIST
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.
FDA Approves Atezolizumab Monotherapy in Advanced PD-L1-High NSCLC
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.
Trastuzumab Deruxtecan Receives Orphan Drug Designation from FDA for Gastric Cancer
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.
FDA Approves Brigatinib as Frontline Treatment of ALK-Positive Metastatic NSCLC
On May 22, 2020, the FDA has granted approval to brigatinib (Alunbrig) for the frontline treatment of patients with ALK-positive metastatic NSCLC, as detected by an FDA-approved test.
FDA Approves Frontline Nivolumab/Ipilimumab/Chemo for Advanced NSCLC
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.
FDA Cancels ODAC Meeting for BLA of Margetuximab in HER2+ Breast Cancer
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.
Onvansertib Receives FDA Fast Track Designation in KRAS+ mCRC
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.
FDA Approves Atezolizumab Combo for Unresectable or Metastatic Hepatocellular Carcinoma
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 Demonstrates Significant Findings in RET+ Solid Tumors as FDA Grants Priority Review
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.
FDA Approves Frontline Ramucirumab/Erlotinib for EGFR+ mNSCLC
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.