Capricor Therapeutics Inc (NASDAQ:CAPR) saw its share price jump 14.3% on Tuesday following an announcement that the U.S. Food and Drug Administration has lifted a Complete Response Letter and resumed its review of the companys Biologics License Application for Deramiocel, an investigational cell therapy for cardiomyopathy associated with Duchenne muscular dystrophy.
In regulatory guidance tied to the resubmission, the FDA classified the filing as a Class 2 resubmission and assigned a Prescription Drug User Fee Act target action date of August 22, 2026. According to the companies announcement, the agency has not identified any potential review issues in its response to the resubmitted application.
The BLA rests on results from the pivotal HOPE-3 Phase 3 trial, which the company reports met the primary endpoint and all Type I error-controlled secondary endpoints. Capricor and its supporters characterize Deramiocel as having the potential to become the first therapy to treat both the skeletal and cardiac manifestations of Duchenne muscular dystrophy.
Capricor previously received a Complete Response Letter from the FDA in July 2025. After submitting additional data and supporting documentation from the HOPE-3 clinical study, the company said the FDA has resumed its review of the application.
The company also noted it expects to be eligible for a Priority Review Voucher upon potential approval of Deramiocel. In February 2026, Congress extended the Rare Pediatric Disease Priority Review Voucher program through September 30, 2029, a legislative update the company referenced in communications about the regulatory pathway.
Market and analyst reactions
Market response was immediate, with CAPR shares advancing double digits on the news. Analysts provided context for the companies regulatory progress. H.C. Wainwright analyst Joseph Pantginis highlighted that Capricor had submitted additional data and supporting documentation from the HOPE-3 Phase 3 study as part of its response to the CRL, and that management reports the FDA has not identified any potential review issues in its response to the resubmission.
Jones Trading analyst Catherine Novack emphasized a different factor, noting that the departure of the CBER head Dr. Vinay Prasad reduced a source of regulatory risk. Novack characterized Prasads prior actions as erratic and cited his vocal opposition to Deramiocel as having added regulatory uncertainty prior to his departure.
Impacted sectors
- Biotechnology and biopharmaceutical companies focused on rare diseases and cell therapies
- Healthcare investors tracking regulatory outcomes and drug approval catalysts
- Markets for priority review vouchers and pediatric rare disease policy
What remains uncertain
- Final FDA action: although review has resumed and no review issues were identified in the agencies response to the resubmission, the outcome of the regulatory review remains pending until the target action date.
- Market and clinical adoption: potential clinical adoption and commercial performance are contingent on an approval decision and are not resolved by the resumed review.