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June 24, 2025 / Advocacy,Research

Capricor Shares Regulatory Update on Deramiocel BLA for Duchenne

Capricor Therapeutics has shared a regulatory update related to its Biologics License Application (BLA) for Deramiocel, the company’s novel cell therapy for the treatment of Duchenne-associated cardiomyopathy.

As part of the FDA’s ongoing review, Capricor has been informed that an Advisory Committee (AdComm) meeting is not indicated at this time, and the previously scheduled meeting has been canceled. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Capricor indicated that to date, all regulatory milestones have proceeded as expected, including a successful pre-license inspection and a mid-cycle review with no major issues, and an in-person late-cycle review meeting remains scheduled for mid-July. 

Four-year data from Capricor’s HOPE-2 Open-Label Extension (OLE) study was recently presented at PPMD’s 2025 Annual Conference, demonstrating continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment. Conference attendees can access a recording of this session through the Conference Hub now. 

PPMD recognizes that this decision by the FDA to cancel the AdComm may create uncertainty within our community. We are working with advocacy partners and continue to actively engage with the FDA and the community to ensure that our collective voices and real-world experiences are part of the review process. We remain unwavering in our commitment to amplify your voices and keep the community involved and informed every step of the way.

Read Capricor’s press release here.

Read Capricor’s community letter here.

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