FDA panel to consider previously spurned drugs
The Food and Drug Administration is shifting its focus this week toward reviewing medical therapies that previously faced rejection. An advisory committee will meet for two days to reconsider applications for drug candidates that lost support under former agency leadership. This process represents a significant moment for the companies involved as they argue that existing clinical data warrants a closer look.
On Wednesday, the Cellular, Tissue and Gene Therapy Advisory Committee examines Capricor’s deramiocel, a cell therapy intended for Duchenne muscular dystrophy. While FDA briefing documents suggest skepticism regarding the efficacy data, Capricor maintains that newer cohorts show meaningful results for patients. The discussion will highlight disagreements over statistical methods and the timing of trial unblinding.
Thursday shifts to Replimune’s immunotherapy for melanoma. This treatment faced past rejections, and the upcoming meeting serves as a high-stakes test for the company’s regulatory strategy. These reviews occur amid broader agency discussions regarding pharmacy compounding and clinical trial access.
Separately, the FDA is addressing systemic issues in cancer research. New guidance documents aim to expand patient participation in clinical trials by relaxing eligibility criteria. Current data shows fewer than five percent of cancer patients enroll in trials despite high interest, a gap the agency hopes to close by updating standards for laboratory values and medication exclusions.