FDA Says Capricor Duchenne Drug Missed Phase 3 Goals
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- FDA said Monday that Capricor Therapeutics' stem cell drug deramiocel did not meet the objectives of its Phase 3 trial for Duchenne muscular dystrophy.
- Capricor Therapeutics said in December that deramiocel met both primary and secondary endpoints in a large, randomized study — a claim the FDA now publicly disputes.
- Deramiocel was tested primarily in teenagers and young men who had already lost the ability to walk, a population with few treatment options.
- Capricor had said the drug appeared to preserve upper-arm function and delay the heart failure most Duchenne patients eventually experience.
- The FDA position comes ahead of an advisory hearing that will evaluate whether the drug should be approved.
Why it matters: The FDA's public skepticism, released days before an advisory committee hearing, directly contradicts Capricor's December announcement of trial success and puts the company's path to approval in jeopardy for a fatal childhood disease with no good treatment options for non-ambulatory patients.
