FDA briefing hints tough road for Capricor and Replimune ahead of advisory committee meetings

The FDA released briefing documents for upcoming advisory committee meetings concerning Capricor Therapeutics' Duchenne muscular dystrophy cell therapy and Replimune's advanced melanoma candidate.

In the Capricor briefing, the agency challenged the company's claim that its Phase 3 trial met primary endpoints, suggesting the data may not support approval. For Replimune, the FDA expressed concerns about the reliance on a single‑arm study to demonstrate efficacy.

Experts note that the resumption of advisory committee meetings, after a pause under the previous commissioner, is unexpected given staffing constraints, adding uncertainty to the review process.

The developments come as GSK and AstraZeneca present ambitious Q2 plans and Sarepta announces a new CEO, but the regulatory outlook for the two smaller biotechs appears uncertain.