Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, has provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy.

As part of the FDA’s ongoing review, the company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.

Linda Marbán, Chief Executive Officer of Capricor said, “We remain confident in the strength of our submission and continue to advance toward potential approval, with our next major step being the upcoming late-cycle review meeting.”

She further added, “To date, all regulatory milestones have proceeded as expected, including a successful pre-license inspection and a mid-cycle review with no major issues. Our application remains under Priority Review, and we believe we are well-positioned as we move toward our PDUFA date. We continue to work closely with the FDA and remain encouraged by the progress of our review.”

Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function. The data demonstrated continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment.