FDA Advisory Committee Votes Against Capricor's Deramiocel in DMD Cardiomyopathy; Review Extended to November 2026
On July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 (with no abstentions) that the available clinical evidence does not support the effectiveness of Capricor Therapeutics' investigational cell therapy, deramiocel (CAP-1002), for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).
However, in a major regulatory update on August 24, 2026, the FDA extended the Prescription Drug User Fee Act (PDUFA) target action date for the deramiocel Biologics License Application (BLA) from August 22, 2026, to November 22, 2026. This three-month extension was triggered by Capricor's submission of a major amendment to its application under FDA guidance.
Strategic Shift in Regulatory Focus
Following the highly critical Advisory Committee meeting, Capricor pivoted its regulatory strategy to salvage the BLA:
- Indication Narrowing: Capricor shifted the primary focus of its submission from DMD cardiomyopathy (the subject of the negative vote) to upper limb function in DMD. Because the advisory committee never formally voted on whether deramiocel improves upper limb function, this shift effectively bypasses the committee's negative verdict on the cardiac endpoint.
- Additional Clinical Data: The major amendment accepted by the FDA includes newly available long-term data from Capricor's Phase 3 HOPE-3 clinical trial (NCT05126758), as well as additional statistical analyses. These clinical details provide an extra year of patient follow-up that was unavailable during the initial Advisory Committee review.
Ongoing Clinical and Regulatory Debate
While the extension provides a critical lifeline for deramiocel, significant regulatory risks remain:
- During the July Advisory Committee briefing, the FDA noted that Capricor's Phase 3 trial had failed to show a statistically significant or clinically meaningful effect on its pre-specified upper limb function endpoint.
- While Capricor presented a post-hoc statistically significant analysis of the primary upper limb endpoint, the FDA concluded the results were difficult to interpret.
- Multiple independent experts on the advisory panel expressed skepticism regarding the upper limb function data, with some noting that a real effect on muscle strength was difficult to discern from the limited sample size.
The FDA now has until November 22, 2026, to assess whether the newly submitted long-term HOPE-3 data, combined with the original clinical evidence, is sufficient to support approval for the revised upper limb function indication.