STAT+: FDA expert panel recommends against approval of Capricor’s Duchenne drug
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- FDA expert panel voted 9-3 that Capricor Therapeutics' deramiocel should not be approved for Duchenne muscular dystrophy.
- The panel concluded the clinical data for deramiocel did not provide 'substantial evidence' of efficacy.
- The voting question specifically centered on whether deramiocel could treat Duchenne-related cardiomyopathy.
- FDA is not required to follow the panel's recommendation but typically does, according to the source.
- The agency review is still underway with a decision expected by August 22.
Why it matters: The 9-3 panel vote against deramiocel, from a body the FDA usually follows, leaves Capricor's Duchenne cardiomyopathy treatment facing steep odds when the agency rules by August 22 — denying families and investors the therapy's commercial and clinical path.
