Capricor Therapeutics has submitted a Biologics License Application (BLA) to the U.S. FDA for deramiocel, a cell therapy designed to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. This submission triggered a $10 million milestone payment from Nippon Shinyaku, Capricor’s distribution partner. If approved, deramiocel would be the first therapy specifically targeting DMD-related heart disease.
This BLA submission is a critical advancement in the fight against DMD, a devastating genetic disorder affecting approximately 15,000-20,000 individuals in the U.S. Current treatment options for DMD are limited, and there is no cure. DMD cardiomyopathy, a debilitating heart condition caused by the disease, is the leading cause of death among DMD patients. Deramiocel offers a potential breakthrough for these patients, addressing the critical unmet need for a therapy that specifically targets this life-threatening complication. The therapy’s potential to slow or reverse cardiac damage could significantly improve the quality of life and potentially extend the lifespan of DMD patients.
The BLA submission is based on positive data from Capricor’s Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials. These data, compared to natural history data on DMD cardiomyopathy progression, demonstrated the potential of deramiocel to attenuate the cardiac implications of the disease. Capricor has requested priority review, which could shorten the FDA review process to six months. Deramiocel has already received Orphan Drug Designation from both the FDA and the European Medicines Agency, underscoring its potential to address an unmet medical need. Furthermore, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. and Advanced Therapy Medicinal Product (ATMP) designation in Europe, expediting the development and review processes. Should deramiocel receive FDA approval, Capricor would be eligible for a Priority Review Voucher, a valuable asset that can be used to expedite the review of a future drug application.
The BLA submission represents a major milestone for Capricor and a significant step towards potential approval of the first therapy for DMD cardiomyopathy. A positive FDA decision would validate years of research and development, potentially transforming the treatment landscape for DMD patients suffering from this life-threatening heart condition. It would also position Capricor as a leader in the field of regenerative medicine and pave the way for further development of its innovative cell and exosome-based therapies for other rare diseases. Pending approval, the next steps will include finalizing commercialization plans with Nippon Shinyaku and preparing for the potential launch of this much-needed therapy.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
