Committee says company didn’t provide ‘substantial evidence’ of efficacy
An image of tissue from a patient with Duchenne muscular dystrophy.Dr. Edwin P. Ewing, Jr./CDC
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.Andrew covers the biopharma industry, scientific research, and public health across the continent. You can reach Andrew confidentially on Signal at drewqjoseph.71.
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.
