FDA Advisory Panel Votes Against Duchenne Muscular Dystrophy Heart Treatment, Setting Up August Deadline
An FDA advisory committee voted 9–3 that evidence was insufficient to establish the effectiveness of Deramiocel, Capricor Therapeutics' treatment for a heart condition associated with Duchenne muscular dystrophy (DMD). The decision can be reversed before an Aug. 22 deadline. The vote follows a prolonged regulatory process in which the FDA declined to approve Deramiocel, later reopened its application after receiving additional data, and then convened the panel that returned the negative vote.
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The FDA's advisory committee voted 9–3 against recommending approval of Deramiocel, a Capricor Therapeutics treatment targeting the cardiac complications of Duchenne muscular dystrophy, determining that available evidence was insufficient to establish effectiveness. Patients and advocates now face an Aug. 22 deadline for that decision to be reversed.
The Washington Examiner op-ed, authored by Tricia Flanagan — founder and CEO of Azuza Laboratories and a rare disease advocate — described the FDA's handling of Deramiocel as a "bureaucratic ordeal," citing the agency's sequence of declining the application, reopening it after additional data, and then convening a panel that asked for still more evidence. Flanagan argued this sequence reflects shifting evidentiary standards rather than a stable, transparent approval pathway.
The piece drew a broader argument about consistency in rare-disease regulation, noting that companies in the Huntington's disease space and those developing cell and gene therapies have similarly described encountering new standards late in the approval process after investing years following earlier FDA guidance. Flanagan stated that predictable standards are a precondition for medical innovation.