FDA advisory committee meetings sow confusion, threaten trust with rare disease developers

The Food and Drug Administration has been working to repair its relationship with rare-disease drug makers and patient groups since former commissioner Marty Makary left three months ago.

Last week, advisory committee sessions for Replimune and Capricor Therapeutics added new uncertainty, with participants describing the process as chaotic and frustrating.

In the Capricor case, the Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 against recommending approval of the Duchenne muscular dystrophy cell therapy deramiocel. A mother of a trial participant criticized the FDA’s analysis as disingenuous and noted a decline in her son’s heart function before treatment.

Analysts say the outcome could erode the goodwill the agency has been trying to rebuild with the rare-disease community and may affect future collaborations and submissions.