Capricor Therapeutics Responds After FDA Publicly Posts Complete Response Letter For Deramiocel, Its Investigational DMD Cell Therapy

Capricor Therapeutics, a biotechnology company advancing cell and exosome-based therapies for rare diseases, has issued a statement regarding the U.S. Food and Drug Administration’s (FDA) recent public posting of its Complete Response Letter (CRL) for Deramiocel. Deramiocel is the company’s investigational cell therapy designed for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The company explained that it was not informed beforehand that the FDA would make the CRL publicly available. The letter, which was originally received in July 2025, outlines the agency’s feedback on the Biologics License Application (BLA) for Deramiocel. While the FDA has chosen to publish the letter, it did not release Capricor’s preliminary written response, which was submitted shortly after receipt of the CRL. In that response, the company addressed points raised by the agency, provided clarifications, and presented its plan to resolve the outstanding issues.

Linda Marbán, Ph.D., Chief Executive Officer, stated, “Transparency is vital in regulatory communications, especially when patients are waiting for therapies with the potential to alter the course of devastating diseases such as Duchenne muscular dystrophy. Our focus remains on working closely with the FDA to resolve the outstanding issues and to advance Deramiocel toward approval. While we respect the FDA’s process, we believe it is important that the public has visibility into both the CRL and our detailed written response submitted to the Agency.”

He also added, “We are now awaiting the official minutes from our recent Type A meeting with the FDA review team, expected to be issued later this quarter, which will help define the next steps in our regulatory pathway. Looking ahead, we expect topline HOPE-3 data in the fourth quarter of 2025, and our discussions with the FDA have centered on how these data will inform and support the timing of our BLA resubmission.”

To ensure transparency for patients, families, and other stakeholders, Capricor announced that it will publish its full preliminary response on the investor section of its website. This step is intended to provide clear visibility into the company’s communications with the FDA and its strategy for advancing the Deramiocel program.