Capricor Therapeutics Begins Rolling BLA for Duchenne Therapy Deramiocel

Capricor Therapeutics has initiated the rolling submission of a Biologics License Application (BLA) to the FDA for its cell-based therapy, deramiocel, as a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The company plans to complete the submission by year-end 2024, with the possibility of FDA Priority Review. This submission represents a significant step in obtaining full approval for deramiocel, a potential breakthrough for patients with this rare disease.

Capricor Therapeutics (NASDAQ: CAPR), a pioneering biotechnology firm specializing in transformative cell and exosome-based therapies for rare diseases, recently announced the initiation of a rolling submission process for a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for its leading cell-based therapy, deramiocel [1]. This development brings Capricor closer to obtaining full approval for deramiocel as a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy, a potentially groundbreaking milestone for patients with this rare and debilitating condition.

DMD, a genetic disorder characterized by progressive muscle wasting and weakness, can lead to severe cardiac complications, including cardiomyopathy [2]. Currently, there are no approved treatment options available for DMD cardiomyopathy. Deramiocel, an allogeneic population of cardiosphere-derived cells (CDCs), has shown promising results in preclinical and clinical studies, demonstrating potent immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure [1].

Capricor plans to complete its rolling BLA submission by the end of 2024. The application may be eligible for priority review due to deramiocel's potential to significantly improve the safety and/or effectiveness of treatment for DMD cardiomyopathy [1]. The company anticipates that the FDA will formally accept the submission for review once it is completed.

Deramiocel has received Orphan Drug Designation and the regulatory pathway is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) [1]. In addition, if Capricor were to receive FDA approval for deramiocel, it would enable the expedited delivery of this novel, first-in-class treatment to patients in need.

This submission marks a significant step forward in the regulatory process for deramiocel, potentially paving the way for its approval as a treatment for DMD cardiomyopathy. With the potential to provide a much-needed treatment option for patients with this rare and life-threatening condition, Capricor's efforts continue to showcase the company's commitment to transforming lives through innovative therapeutic solutions.

References:

[1] Capricor Therapeutics. (2024, October 9). Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application (BLA) with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy. https://www.globenewswire.com/news-release/2024/10/09/2960610/0/en/Capricor-Therapeutics-Announces-Initiation-of-Rolling-Submission-of-Biologics-License-Application-BLA-with-U-S-FDA-for-Deramiocel-for-the-Treatment-of-Duchenne-Muscular-Dystrophy.html