New FDA Specialty Approvals
✦ Jan. 8: The FDA expanded the label of Keytruda (pembrolizumab) to include the treatment of people with bacillus Calmette-Guérin-unresponsive, high-risk, non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors who are ineligible for or have decided not to undergo cystectomy. The agency initially approved the programmed death receptor-1 (PD-1) inhibitor from Merck & Co., Inc. in 2014 (RSP 9/14, p. 4), and it is approved for more than 20 cancer indications. The recommended dose for the newest indication is 200 mg infused intravenously over 30 minutes every three weeks. GoodRx lists the price of one 100 mg/4 mL vial as more than $9,600. For more information, visit www.keytruda.com.
✦ Jan. 9: The FDA approved Blueprint Medicines Corp.’s Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor harboring a platelet-derived growth factor receptor alpha exon 18 mutation, including PDGFRA D842V mutation, the most common exon 18 mutation. The agency gave the kinase inhibitor breakthrough therapy, fast track and orphan drug designations. Tablets are available in 100 mg, 200 mg and 300 mg strengths, and dosing is 300 mg once daily. The company priced all three strengths at $32,000 per 30 days. Visit https://ayvakit.com.