FDA questions the efficacy of Capricor's Duchenne drug
STAT • • business
Key Points:
- The FDA announced that Capricor Therapeutics’ stem cell treatment, deramiocel, for Duchenne muscular dystrophy failed to meet Phase 3 trial objectives, contradicting the company’s previous claims.
- In December, Capricor had reported that deramiocel met both primary and secondary endpoints in a large randomized study, offering hope for this fatal childhood disease.
- The treatment was notable for its effects on teenagers and young men who had lost the ability to walk, potentially preserving upper-arm function and delaying heart failure.
- Despite initial optimism, the FDA’s assessment raises questions about the drug’s efficacy and future prospects for approval.
