Capricor Therapeutics likely faces FDA rejection of its Duchenne cell therapy

Capricor Therapeutics is working to avoid an imminent FDA rejection of its cell‑based treatment for Duchenne muscular dystrophy. The company says it remains confident in the drug, but the regulator’s decision appears near certain.

To keep the program alive, Capricor plans to submit data from an open‑label extension of its previously failed Phase 3 trial. The additional results are intended to demonstrate continued safety and potential benefit.

Analysts describe the move as a stall tactic, noting that the only realistic path to approval would be a new, randomized clinical trial. Without such a study, the therapy, known as deramiocel, is unlikely to meet regulatory standards.

The situation underscores the challenges of advancing cell therapies for rare diseases, especially when early trials do not meet efficacy endpoints.