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Getting the latest healthcare news for you
Getting the latest healthcare news for you
The FDA has extended its review of Capricor's experimental cell therapy, deramiocel, for Duchenne muscular dystrophy by three months to November 22. This follows a rocky regulatory journey — including an FDA advisory panel voting 9-3 against the drug's effectiveness data in July. The extension, driven by new 24-month clinical trial data, has investors cautiously optimistic, with shares surging over 20%.
Capricor Therapeutics' experimental cell therapy, deramiocel, for Duchenne muscular dystrophy (DMD) has been given more time at the FDA's table. The agency extended its review deadline by three months to November 22, 2026, to evaluate new clinical trial data submitted by the company — sending shares up more than 20%.
The road here has been bumpy. In July, an FDA advisory panel voted 9-3 against deramiocel's effectiveness data, echoing staff concerns that Capricor had changed how it measured trial outcomes after the study ended — switching from a 42-point arm-function score to a percentage-change calculation. Reviewers also questioned whether enrolled patients actually had DMD-related cardiomyopathy, given their normal heart function at baseline.
Capricor responded by pivoting its focus to upper limb muscle function — an area where some panel members had been more receptive — and submitted 24-month follow-up data from its HOPE-3 trial.
Key Takeaways:
Why it matters: DMD is a rare, progressive genetic disease affecting mostly boys, with no approved treatment for its associated heart complications. A successful approval of deramiocel could be a landmark moment for this underserved patient population.
