After a major regulatory breakthrough, Capricor Therapeutics, Inc. (NASDAQ: CAPR) stock value has significantly increased. CAPR shares have increased 10.46% to $15.10 as of the most recent premarket check, indicating that investors were optimistic about the company’s recent developments.
FDA Accepts Biologics License Application
The U.S. Food and Drug Administration (FDA) has accepted Capricor’s Biologics License Application (BLA) for deramiocel, an investigational cell therapy that targets the cardiomyopathy associated with Duchenne muscular dystrophy (DMD), for review. This is a big step in the direction of the therapy’s potential full approval.
Additionally, the FDA granted the application Priority Review, setting August 31, 2025, as the target action date under the Prescription Drug User Fee Act (PDUFA). As of now, the FDA has not discovered any issues that would prolong the review procedure.
Potential Progress in DMD-Cardiomyopathy Management
Capricor is one step closer to creating a revolutionary treatment for DMD-cardiomyopathy, a condition for which there are currently no approved therapies, thanks to the BLA’s acceptance. With the potential for broad use throughout the DMD-cardiomyopathy therapy landscape, deramiocel is anticipated to be a quarterly, lifetime medication if authorized.
Clinical data from Capricor’s Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) studies, which have shown encouraging results when compared to an FDA-funded dataset on the development of DMD cardiomyopathy, support the application.
Regulatory Labels Boost the Chance of Approval
One of the main causes of death for DMD patients, DMD-cardiomyopathy, has been effectively mitigated with deramiocel. The possible approval of this medicine might mark a major improvement in available treatment alternatives due to the encouraging safety and effectiveness evidence. Deramiocel’s potential to enhance patient outcomes is highlighted by its FDA Priority Review status.
Further highlighting the therapy’s significance is the fact that it has been designated an Orphan Drug by the European Medicines Agency (EMA) and the FDA. Capricor’s RMAT (Regenerative Medicine Advanced Therapy) and ATMP (Advanced Therapy Medicinal Product) designations in the US and Europe, respectively, support its regulatory approach.
Additionally, because of its prior categorization as a rare pediatric condition, Capricor may be eligible for a Priority Review Voucher (PRV) if the FDA approves commercialization by September 30, 2026.
