Today, Capricor Therapeutics shared an important update on the FDA’s review of the Biologics License Application (BLA) for Deramiocel, the company’s cell therapy for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy.

Capricor has indicated that the company plans to submit an amendment to the FDA that will include additional data and analyses to support the ongoing review of Deramiocel. FDA has suggested to Capricor that once the amendment is submitted and received by the agency, the current Prescription Drug User Fee Act (PDUFA) action date of August 22, 2026, will be extended. 

Capricor has confirmed that the BLA amendment will include the 24-month Open Label Extension (OLE) data from the HOPE-3 trial and additional distal analyses from the existing data package. The additional analyses represent a narrowing of the indication to upper limb dysfunction only.

PPMD appreciates FDA’s willingness to accept additional information and analysis for consideration as part of its review. Given the questions raised during the July 29, 2026, FDA Advisory Committee meeting, it is important that the additional analyses are completed and evaluated through the regulatory process. 

What Happens Next? 

Capricor did not indicate whether its BLA amendment submission would be designated a major amendment. A non-major amendment does not carry the same defined regulatory timeline that would apply to a major amendment. With this, it remains unclear how long the PDUFA date may be extended.

It is important to note that the submission of an amendment does not indicate what the FDA’s ultimate decision will be. FDA will determine whether the totality of the evidence supports regulatory action after completing its review.

PPMD will continue to call for a comprehensive and timely review. For the Duchenne community, time matters. This regulatory process is not yet complete, and we encourage the community to stay engaged and informed as we move through the next steps. We will continue to share updates as they become available.

Read Capricor’s press release here.