STAT+: FDA expert panel recommends against approval of Capricor’s Duchenne drug

A group of outside experts convened Wednesday by the Food and Drug Administration concluded clinical data for a treatment for Duchenne muscular dystrophy from Capricor Therapeutics did not provide “substantial evidence” of efficacy.  

The vote was 9-3, sending to the FDA a recommendation that the Capricor treatment, called deramiocel, should not be approved. 

The FDA is not required to adhere to the expert panel’s stance, but it typically does. The agency review is still underway with a decision expected by Aug. 22. 

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