Capricor's Duchenne Drug Deramiocel Headed for FDA Rejection
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- Capricor Therapeutics is trying to stave off a near-certain FDA rejection of deramiocel, its cell therapy for Duchenne muscular dystrophy
- The company's strategy is submitting results from an open-label extension of its failed Phase 3 study
- The source characterizes the move as a "stall tactic" and "nothing more"
- Per the source, the only path to approval for deramiocel is an entirely new, randomized clinical trial
Why it matters: Duchenne muscular dystrophy patients waiting on a new therapy face continued delay — the resubmitted extension data doesn't fix the underlying problem (the Phase 3 already failed), and the FDA will require a brand-new randomized trial before considering approval.
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