publication date: Dec. 20, 2019

Pazdur on the challenge of approving drugs for small indications

ODAC recommends approval for tazemetostat in rare sarcoma, sNDAs for Lynparza, Keytruda

By Paul Goldberg

The FDA Oncologic Drugs Advisory Committee in a session Dec. 17 and 18 considered three applications that were arranged thematically, to show how the agency is approaching indications where the number of patients is small and the disease catastrophic.

In such indications, a demand for two randomized trials, or even one randomized trial, showing overall survival would mean that no therapies will get to patients—ever.

During this unusually long two-day session, the committee that advises FDA on clinical matters recommended approval for tazemetostat, a drug for the treatment of an extremely rare sarcoma, as well as two new indications for Lynparza in a pancreatic cancer indication and Keytruda in a bladder cancer indication.

But to anyone interested in drug development, the main take-away would be remarks by FDA’s cancer czar Richard Pazdur on the challenges of approving drugs in the era of ever-splintering indications.

ODAC voted 7:5 to recommend approval for Lynparza (olaparib) for the maintenance treatment of adult patients with deleterious or suspected deleterious gBRCAm metastatic adenocarcinoma of the pancreas whose disease has not progressed on first-line platinum-based chemotherapy. The agent is sponsored by AstraZeneca. The briefing document is posted here.

In a 9:4 vote, the committee recommended approval of Keytruda (pembrolizumab) for patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer with carcinoma in-situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy. Keytruda is sponsored by Merck Sharp & Dohme Corp. The briefing document is … Continue reading Pazdur on the challenge of approving drugs for small indications

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