FDA questions efficacy of Capricor's Duchenne muscular dystrophy cell therapy ahead of advisory committee

FDA reviewers have raised concerns about the efficacy of Capricor's cell therapy for Duchenne muscular dystrophy as the agency prepares for an advisory committee meeting. The agency said it could not find sufficient evidence that the treatment improves clinical outcomes, and it has asked the company for additional data.

The lack of clear efficacy data has led to a sharp drop in Capricor's share price, reflecting heightened investor uncertainty. The comments also increase doubt among patients and clinicians who were hoping for a new therapeutic option.

The advisory committee is slated to meet on Wednesday, where the FDA will discuss the data and decide on the next steps for the therapy's development.