FDA rejects Capricor’s Duchenne cell therapy

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.

The Food and Drug Administration rejected a marketing application from Capricor Therapeutics for a cell therapy to treat Duchenne muscular dystrophy, the company said Friday. 

In its letter to Capricor, the FDA said the company’s application “does not meet the statutory requirement for substantial evidence of effectiveness” and requested additional clinical data, the company said. 

Capricor submitted a marketing application to the FDA at the end of December. Its off-the-shelf cell therapy, called deramiocel, would have been the first treatment cleared specifically for the cardiomyopathy, a serious heart condition, that is associated with Duchenne. It could have also been used alongside other Duchenne drugs or gene therapies.