Capricor Therapeutics Submits BLA for FDA Approval of Deramiocel in DMD

Capricor Therapeutics, a biotech firm focused on developing innovative cell and exosome-based therapies for rare diseases, has announced the completion of the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for full approval of deramiocel, an investigational cell therapy aimed at treating patients with Duchenne muscular dystrophy (DMD) cardiomyopathy.

“The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need,” said Linda Marbán, Ph.D., chief executive officer of Capricor. “We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval.”

The complete filing of the rolling BLA was finalized as the company had earlier indicated in late December 2024 and is backed by Capricor’s current cardiac findings from its phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, relative to natural history data from an FDA-funded and published dataset regarding the effects of DMD cardiomyopathy and possible biomarkers of disease progression. Capricor has sought a priority review, which, if approved, would shorten the review period from the usual 10 months to a prioritized 6-month timeline starting from when the FDA accepts the submission.