FDA Approvals, News & Updates

On January 8, 2020, the FDA accelerated the approval of a new indi­cation for pembrolizumab (Keytruda; Merck & Co) for the treatment of Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk, non–muscle invasive bladder cancer and carcinoma in situ, with or without papillary tumors, in patients who are ineligible for or who have elected not to undergo cystectomy. Pembrolizumab has been previously approved for many indications.

On December 23, 2019, the FDA accelerated the approval of fam-trastuzumab deruxtecan-nxki (Enhertu; Daiichi Sankyo), a HER2-directed antibody and topoisomerase inhibitor conjugate, for the treatment of adults with unresectable or metastatic HER2-positive breast cancer after ≥2 previous anti-HER2–based regimens in the metastatic setting. The FDA granted fam-trastuzumab deruxtecan-nxki a breakthrough therapy designation.

On January 9, 2020, the FDA accelerated the approval of avapritinib (Ayvakit; Blueprint Medicines Corporation), a kinase inhibitor, for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. This approval includes GIST that harbors a PDGFRA D842V mutation, which is the most common exon 18 mutation. The FDA granted breakthrough therapy and orphan drug designations to avapritinib.

On January 23, 2020, the FDA accelerated the approval of tazemetostat (Tazverik; Epizyme Inc), an EZH2 methyltransferase blocker, for the treatment of patients aged ≥16 years with metastatic or locally advanced epithelioid sarcoma (a subtype of soft-tissue sarcoma) that is not eligible for complete resection. Tazemetostat received an orphan drug designation for this indication.

On December 18, 2019, the FDA accelerated the approval of enfortumab vedotin-ejfv (Padcev; Astellas Pharma), a Nectin-4–directed antibody and microtubule inhibitor conjugate, for the treatment of adults with locally advanced or metastatic urothelial cancer after immunotherapy with a PD-1 or PD-L1 inhibitor and a platinum-containing chemotherapy. These are the current standard treatments for patients with bladder cancer, the sixth most common cancer in the United States. Urothelial cancer accounts for >90% of bladder cancers.

On January 29, 2020, Eli Lilly announced that the FDA granted priority review to their New Drug Application for selpercatinib (LOXO-292) for the treatment of patients with advanced RET fusion-positive non–small-cell lung cancer, RET-mutant medullary thyroid cancer, and RET fusion-positive thyroid cancer.

On January 23, 2020, the FDA granted accelerated approval to tazemetostat (Tazverik; Epizyme), a methyltransferase inhibitor, for the treatment of metastatic or locally advanced epithelioid sarcoma in adults and pediatric patients aged ≥16 years who are not eligible for complete resection.

On January 8, 2020, the American Cancer Society published details of their annual cancer statistics report, including the most recent data on population‐based findings for cancer incidence through 2016 and for mortality through 2017 (CA Cancer J Clin. 2020;70:7-30). The report revealed encouraging news as well as some sobering trends.

The FDA approval of avapritinib marks the first time a drug has been approved specifically for patients with GIST harboring PDGFRA exon 18 mutations, which are involved in approximately 10% of GIST cases.

The FDA has confirmed another strong year regarding the approval of innovative new drugs and biosimilars, including several agents used in the treatment of patients with cancer.