Capricor Submits Biological License Application For Deramiocel

Capricor Therapeutics, a biotechnology company focused on innovative cell and exosome-based therapies for rare diseases, has completed the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). The application seeks full approval for deramiocel, an investigational cell therapy designed to treat cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).

The submission, completed as planned in late December 2024, is backed by strong cardiac data from Capricor’s Phase 2 HOPE-2 trials and its Open Label Extension (OLE) study. This data was compared against natural history data from an FDA-funded dataset that explores DMD cardiomyopathy and biomarkers related to disease progression. Capricor has also requested a priority review from the FDA, which could shorten the review process from the standard 10 months to just 6 months if granted.

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, said, “The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. This BLA is the culmination of a body of work focused on bringing this potentially transformational therapy to those patients in need. We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval.”

In recognition of this milestone, Capricor will receive a $10 million payment from its U.S. commercialisation and distribution partner, Nippon Shinyaku Co., Ltd., under their existing agreement. Deramiocel has previously been granted Orphan Drug Designation by the FDA and the European Medicines Agency (EMA). Its regulatory pathway is further supported by RMAT (Regenerative Medicine Advanced Therapy) Designation in the U.S. and ATMP (Advanced Therapy Medicinal Product) Designation in Europe. If the FDA grants marketing approval, Capricor will qualify for a Priority Review Voucher (PRV) due to Deramiocel’s rare pediatric disease designation.