Capricor Therapeutics Submits Biologics License Application for Deramiocel to FDA for Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology firm based in San Diego, has recently completed the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). This application seeks full approval for their investigative therapy, deramiocel, aimed at treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD). If approved, deramiocel would mark a significant milestone as it would be the first therapy sanctioned for this specific condition.

The submission represents a significant achievement for Capricor and individuals affected by DMD, as highlighted by the company's CEO, Linda Marbán. She emphasized the potential impact of deramiocel in mitigating the cardiac symptoms related to DMD, showcasing promising results in various clinical trials. The company is eager to collaborate with the FDA to facilitate the review process and support the potential approval of this innovative therapy.

Capricor's BLA submission, completed at the end of December 2024, is built upon extensive cardiac data derived from their Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials. It also includes comparisons to natural history data funded and published by the FDA, which examine the progression of DMD cardiomyopathy and associated biomarkers. Capricor has requested a priority review from the FDA, which, if granted, would expedite the review period from the standard ten months to six months.

In conjunction with this regulatory milestone, Capricor is set to receive a $10 million milestone payment from Nippon Shinyaku Co., Ltd., under their U.S. Commercialization and Distribution Agreement. Deramiocel has garnered Orphan Drug Designation from both the FDA and the European Medicines Agency, bolstering its regulatory pathway. It is also supported by the Regenerative Medicine Advanced Therapy Designation in the U.S. and the Advanced Therapy Medicinal Product Designation in Europe. Moreover, if deramiocel secures FDA marketing approval, Capricor may be eligible for a Priority Review Voucher, owing to its earlier rare pediatric disease designation.

Deramiocel, also known as CAP-1002, is composed of allogeneic cardiosphere-derived cells (CDCs). These cells have shown impressive immunomodulatory, antifibrotic, and regenerative capabilities in both preclinical and clinical settings, specifically targeting dystrophinopathies and heart failure. CDCs operate through the secretion of extracellular vesicles called exosomes, which modulate macrophage expression to promote healing rather than inflammation. This innovative approach has been documented in over 100 peer-reviewed publications, with more than 200 human subjects having participated in clinical trials involving CDCs.

Duchenne muscular dystrophy is a severe genetic disorder that leads to progressive muscle weakness, particularly affecting skeletal, cardiac, and respiratory muscles. The condition typically results in mortality around the age of 30. Affecting approximately one in every 3,500 male births, the patient population in the U.S. is estimated to be between 15,000 and 20,000. The absence of functional dystrophin, a vital structural protein in muscle cells, results in muscle deterioration and fibrotic replacement, eventually leading to cardiomyopathy and heart failure, the leading cause of death in DMD patients. Currently, treatment options for DMD are limited, and there is no cure.

Capricor Therapeutics is committed to advancing groundbreaking cell and exosome-based therapies, with deramiocel at the forefront of its efforts. The company is also exploring exosome technology through its proprietary StealthX™ platform, aimed at developing solutions in vaccinology, and the targeted delivery of various therapeutic agents. Capricor is dedicated to pioneering transformative treatments for those in need, pushing the boundaries of medical science.