An FDA panel said that trial data did not provide substantial evidence showing that investigational deramiocel was effective in treating cardiomyopathy among patients with Duchenne muscular dystrophy.
In a 9-3 vote, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee said deramiocel did not meet the cardiac endpoint, though the primary outcome of the drug's phase III HOPE-3 trial was change in upper limb function.
Much of the meeting focused on how outcomes of the HOPE-3 trial should be interpreted.
Karim Mikhail, BPharm, MSc, acting director of the FDA's Center for Biologics Evaluation and Research, said the meeting focused on cardiomyopathy because drug developer Capricor Therapeutics originally sought that indication in its marketing application for deramiocel.
For both upper limb and heart outcomes, "the data that we have is very fragile," said committee member Steven Pavlakis, MD, of SUNY Downstate Health Sciences University in Brooklyn, New York, who voted against the drug.
