Capricor Therapeutics announced it received a complete response letter (CRL) from US regulator the Food and Drug Administration (FDA) regarding its biologics license application (BLA) for deramiocel (CAP-1002), an exosome-based cell therapy for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The FDA cited insufficient evidence of "substantial efficacy" and unresolved chemistry, manufacturing and control (CMC) issues, though Capricor noted most CMC concerns had been addressed in prior communications. The company plans to submit Phase III HOPE-3 trial data (n=104) in Q3 2025 to strengthen the application.
CAP-1002, derived from cardiosphere-derived cell exosomes containing non-coding RNAs and proteins, showed improved cardiac function and skeletal muscle preservation in 3-year open-label extension data. If approved, it would be the first therapy specifically for DMD cardiomyopathy.
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