FDA Resumes Review of Capricor Therapeutics' Deramiocel Application
The U.S. Food and Drug Administration (FDA) has lifted a prior Complete Response Letter and resumed review of Capricor Therapeutics' Biologics License Application for Deramiocel. A new PDUFA target action date is set for August 22, 2026.
SAN DIEGO – Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has resumed its review of the company's Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA previously issued a Complete Response Letter (CRL) in July 2025.
The agency has now lifted the previous CRL and assigned a new PDUFA target action date of August 22, 2026, for the BLA, classifying the resubmission as Class 2. Capricor Therapeutics expects to be eligible for a Priority Review Voucher upon potential approval.
Capricor CEO Linda Marbán stated the company is encouraged by the FDA's acknowledgment of its response to the CRL and its continued review. The company believes Deramiocel has the potential to be the first therapy to address both skeletal and cardiac manifestations of DMD, supported by positive pivotal HOPE-3 Phase 3 results.
Duchenne muscular dystrophy is a severe, X-linked genetic disorder characterized by progressive muscle degeneration, including cardiac muscle, leading to cardiomyopathy and heart failure as the primary cause of death. Treatment options remain limited, and there is no cure.