FDA Extends Review of Capricor's Deramiocel BLA to November 22 Following Major Amendment and Indication Refinement
The FDA has officially extended its regulatory review of Capricor Therapeutics’ (NASDAQ: CAPR) Biologics License Application (BLA) for deramiocel (CAP-1002) in Duchenne muscular dystrophy (DMD). The new Prescription Drug User Fee Act (PDUFA) target action date has been set to November 22, 2026, representing a three-month extension from the previous August 22, 2026 date. The delay was prompted by Capricor's submission of a major BLA amendment containing newly available clinical data and a refined proposed therapeutic indication.
Background: Advisory Committee Setback and Indication Refinement
The extension follows a challenging regulatory path. Capricor's BLA was originally issued a Complete Response Letter (CRL) in July 2025 due to insufficient efficacy evidence and unresolved Chemistry, Manufacturing, and Controls (CMC) issues. After resubmitting with the completed HOPE-3 Phase 3 clinical trial dataset, the FDA convened an Advisory Committee meeting on July 29, 2026.
The committee voted 9 to 3 against recommending approval, raising concerns about post-randomization changes to the trial's statistical analysis plan and expressing skepticism that left ventricular ejection fraction (LVEF) was a validated surrogate of clinical benefit.
In response to the panel's concerns, Capricor submitted a major BLA amendment that includes:
- Long-Term Data: 24-month open-label extension findings from the HOPE-3 study.
- Additional Analyses: Robustness analyses addressing statistical concerns raised by the FDA.
- Refined Indication: A narrowed proposed indication focusing solely on the preservation of upper limb function—which was the HOPE-3 trial's primary clinical endpoint—rather than broader cardiac/systemic claims.
Clinical Evidence: The HOPE-3 Trial
Despite the negative Advisory Committee vote, the underlying Phase 3 HOPE-3 trial (enrolling 106 boys and young men with DMD, primarily nonambulatory) met its primary and key secondary endpoints:
- Primary Endpoint: A 54% slowing of decline on the Performance of Upper Limb, version 2.0 (PUL v2.0) assessment in the intention-to-treat population (P = .029).
- Key Secondary Endpoint: A 91% slowing of decline in LVEF as measured by centrally read cardiac MRI (P = .041).
- Safety: No unexpected safety signals emerged, with hypersensitivity reactions (the most common adverse events) managed successfully with standard pre-medication.
The FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment and classified it as major. The agency cited the extreme unmet medical need in DMD as a driving factor for continuing to work constructively with Capricor toward a potential approval by the new November 22, 2026 deadline.