FDA Extends Capricor Deramiocel BLA Review

Capricor Therapeutics announced that the FDA has extended the PDUFA target action date for its Deramiocel BLA for Duchenne muscular dystrophy from August 22, 2026 to November 22, 2026 after submitting an amendment with 24-month open-label HOPE-3 data and additional analyses that support a refined indication focused on preserving upper limb function. The extension stems from the FDA classifying the submission as a major amendment requiring more review time, following July 2026 advisory committee discussions. Capricor argues that the extended data strengthen the case for Deramiocel, an allogeneic cell therapy, and highlights its various regulatory designations in the U.S. and Europe, including Orphan Drug and RMAT status. Investor and market coverage notes a mixed backdrop: while clinical progress is positive, the company’s financials show ongoing losses and cash burn, leading some analysts to view CAPR as Neutral for now. Capricor has pointed to the substantial unmet medical need in DMD and the extensive dataset supporting upper limb preservation as key factors behind the refined indication, with the FDA’s review timeline extended to November 2026. The coverage across sources also reflects ongoing investor interest as CAPR trades within a range and continues to be monitored by market alerts.