FDA advisory panel rejects Deramiocel, leaving Duchenne patients facing uncertain future
The FDA’s advisory committee voted 9-3 against approving Deramiocel, a therapy for a heart complication of Duchenne muscular dystrophy, and the decision will stand unless reversed by Aug. 22.
An FDA advisory committee voted 9-3 to reject Deramiocel, Capricor Therapeutics’ candidate for treating a cardiac condition linked to Duchenne muscular dystrophy, and the ruling will remain in effect unless overturned by Aug. 22. The company has endured a protracted regulatory process, including an initial denial, a reopening for additional data, and now this panel’s demand for further evidence. Observers contend that erratic evidentiary thresholds and political risk-aversion hinder access to life-saving therapies for rare-disease patients.
The piece urges the FDA to halt the use of advisory committees until it can guarantee expert composition, clear questions, and consistent standards. It also references the recent departures of FDA commissioner Marty Makary and biologics chief Vinay Prasad amid sustained criticism from patient advocates.
Why it matters
The FDA’s stance could delay a potential treatment for a lethal rare disease, affecting patient survival.
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