FDA Schedules Advisory Panel on Capricor's DMD Cell Therapy BLA

Capricor Therapeutics is a San Diego-based clinical-stage biotechnology company whose entire commercial fate rests on a single asset: Deramiocel, an allogeneic, cardiosphere-derived cell therapy for cardiomyopathy associated with Duchenne muscular dystrophy. DMD is a fatal genetic disease affecting approximately 15,000 people in the United States, primarily boys, with no approved therapy targeting its cardiac manifestations. Cardiomyopathy is the leading cause of death in DMD patients.

Capricor has generated no product revenue since the end of 2024, when it exhausted the recognition of $50 million in milestone payments from its U.S. distribution partner, Nippon Shinyaku. The company lost approximately $105 million in 2025 and $33.9 million in the first quarter of 2026 alone. A December 2025 public offering raised approximately $162 million in net proceeds, leaving the company with roughly $279 million in cash as of March 31, 2026, which management says will support operations into the fourth quarter of 2027.

The company is simultaneously managing a high-stakes regulatory process and active litigation. On May 7, 2026, Capricor filed suit against Nippon Shinyaku and its U.S. subsidiary, NS Pharma, seeking to rescind its U.S. distribution agreement and obtain a preliminary injunction. The complaint alleges a fundamental pricing flaw — tied to the interaction between the contract's pricing formula and Medicare Part B reimbursement rules — that would render distribution of Deramiocel commercially unviable. According to the complaint, once Medicare's average sales price calculation takes effect after the first two quarters of sales, the reimbursement ceiling would make it economically irrational for any party in the supply chain to sell or administer the therapy. Capricor states in the complaint that it accepted NS Pharma's repudiation of the agreement on May 6, 2026.