Capricor's Shares Drop After FDA Rejects Deramiocel Cell Therapy for Heart Disease in DMD

Capricor Therapeutics faced a significant setback on July 11, 2025, when the FDA rejected its cell therapy, Deramiocel, for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The "complete response letter" from the FDA cited the need for more data on the therapy’s efficacy, causing Capricor’s stock to drop by 30%. Deramiocel was developed to address heart complications in DMD, a condition that leads to progressive muscle degeneration, including heart muscle failure.

Deramiocel uses cardiac-derived stem cells to regenerate heart tissue in DMD patients. The therapy involves harvesting stem cells from the patient's own heart, expanding them in the lab, and reintroducing them to the heart to improve its function. The goal is to slow the progression of cardiomyopathy, reducing the need for heart transplants. This approach targets heart muscle regeneration, offering a novel solution for DMD patients, unlike treatments that focus only on muscle degeneration.

The FDA’s decision focused on the lack of sufficient evidence of the therapy’s effectiveness. The agency requested more data to confirm the long-term benefits of the treatment. Capricor is optimistic and plans to resubmit its application in the third quarter, incorporating additional clinical data from an ongoing study. CEO Linda Marban expressed surprise but believes the company can address the FDA’s concerns without needing to submit a completely new Biologics License Application (BLA).

The Deramiocel FDA rejection came earlier than expected, ahead of the FDA's original review date of August 31. Analysts had already raised doubts about approval, citing the cancellation of an advisory panel meeting and the reassignment of key officials at the FDA’s Center for Biologics Evaluation and Research (CBER). Under the leadership of Vinay Prasad, CBER has increased scrutiny on gene and cell therapies, especially regarding accelerated approvals.

Despite the stock drop, Capricor remains committed to advancing Deramiocel. The company hopes to resolve the FDA’s concerns and move forward with its submission. This rejection highlights the challenges biotech companies face in bringing new cell therapies to market, especially for rare diseases like DMD.

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Created: July 11th, 2025

Citations:

Santhosh, C. (2025, July 11). Capricor shares sink as US FDA declines to approve cell therapy. Reuters. https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-declines-approve-capricors-muscle-disorder-therapy-2025-07-11/