On Tuesday, the FDA accepted for review Capricor Therapeutics Inc’s (NASDAQ:CAPR) Biologics License Application for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
DMD cardiomyopathy is a heart condition characterized by cardiac fibrosis, arrhythmias, and heart failure.
Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.
The FDA has not identified any potential review issues.
The BLA submission is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data.
The FDA also informed the company that they have not yet decided whether an Advisory Committee meeting is needed regarding this application.
Deramiocel for DMD has received Orphan Drug Designation from the FDA and European Medicines Agency.
In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD by September 30, 2026, Capricor would be eligible to receive a Priority Review Voucher based on its previous receipt of a rare pediatric disease designation.
Price Action: CAPR stock is up 6.33% at $14.54 at the last check Tuesday.
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