FDA rejects Capricor’s Duchenne cell therapy, citing lack of proven benefit

The FDA rejected Capricor Therapeutics’ application for deramiocel, a cell therapy for Duchenne-related heart issues, citing insufficient evidence of effectiveness and unresolved manufacturing concerns.

Why it matters: Deramiocel was set to be the first treatment for Duchenne cardiomyopathy, a leading cause of death in patients with the disease. The rejection delays a potential breakthrough therapy and highlights stricter regulatory scrutiny.

Backstory:

• Capricor based its application on a small 20-patient Phase 2 trial and historical data, which the FDA deemed inadequate.

• The agency had initially scheduled an advisory committee meeting but later canceled it amid internal leadership changes.

• Capricor claims it followed FDA guidance and plans to present Phase 3 trial results later this year.

Big picture: The rejection reflects a tightening FDA stance on cell and gene therapies. Vinay Prasad, who now oversees such therapies, is known for demanding stronger evidence—a shift from the agency’s previous flexibility. This new regulatory stance could affect future approvals for rare diseases.

What’s next: Capricor shares dropped sharply following the news. A follow-up meeting with the FDA is set, but resubmission could take up to 10 months to review.