FDA Advisory Panel Votes Against Approval of Deramiocel for DMD

An FDA advisory committee voted against recommending approval of Capricor Therapeutics' deramiocel, an allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD), following a contentious meeting dominated by disagreement over which statistical analysis plan (SAP) should govern the pivotal trial results.¹

Reviewers on the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9-3 against approval after a chaotic session that focused heavily on the phase 3 HOPE-3 (NCT00468923) trial's cardiac function data.

Why Did the Committee Vote No?

The committee’s vote was rooted in the fact that the available data did not provide substantial evidence of effectiveness for deramiocel in treating cardiomyopathy in patients with DMD. The discussion centered heavily on the HOPE-3 trial findings, including the interpretation of its cardiac-function findings, missing data, and the statistical methods used to analyze the results.

“When I first read the FDA’s briefing document, parts of it were so hard to reconcile with what actually occurred,” Linda Marban, CEO of Capricor, told the CTGTAC Wednesday morning.²

A major point of disagreement involved the SAP. FDA reviewers based their efficacy assessment in part on an earlier SAP version that they characterized as the prespecified methodology used to design and power the study. Capricor argued that this approach failed to account for subsequent protocol and SAP changes, maintaining that SAP version 3.0, which the company said was finalized before unblinding, was the appropriate basis for interpreting the results.³

The FDA’s concerns extended beyond the SAP dispute. Reviewers questioned the magnitude and consistency of the observed cardiac effects and whether the available data provided sufficiently convincing evidence of clinical benefit.¹ Some panelists viewed the evidence for upper-limb function more favorably than the cardiac findings, but that discussion did not result in a separate affirmative approval recommendation.

“The word of the day seems to be fragile,” Janet Turk Wittes, Wittes, LLC, an expert in randomized controlled trials and a member of the committee, said when explaining why she voted no.² “The results were very fragile. I simply didn’t see enough evidence of benefit to vote yes.”

What Happens Next?

The advisory committee’s recommendation is nonbinding, and the FDA is not required to follow it. The agency’s Prescription Drug User Fee Act (PDUFA) target action date for the deramiocel application is August 22, 2026.

Following the vote, analysts lowered their expectations for approval. Piper Sandler, an American investment bank and financial services company, expects the FDA to issue another complete response letter (CRL) rather than approve deramiocel for DMD cardiomyopathy, although the agency’s final decision remains pending.⁴

This is the second FDA review cycle for deramiocel. In July 2025, the FDA issued a CRL stating that the application did not provide substantial evidence of effectiveness.¹ The agency also identified outstanding chemistry, manufacturing, and controls issues that it said it could not review because of the timing of the CRL.

Capricor subsequently submitted additional clinical data from the phase 3 HOPE-3 trial. The company reported that HOPE-3 met its primary end point, Performance of the Upper Limb version 2.0, as well as the key secondary cardiac end point of left ventricular ejection fraction and other Type I error-controlled secondary end points.⁵

The FDA accepted the company’s resubmission and resumed its review, establishing an August 22, 2026, PDUFA target action date.

Managed Care Implications

DMD-associated cardiomyopathy is a major contributor to morbidity and mortality in Duchenne.¹ Despite this, there is currently no FDA-approved therapy specifically indicated for DMD cardiomyopathy.

If approved, deramiocel would introduce a cell-based treatment option into the DMD care pathway and could raise managed care considerations around coverage criteria, utilization management, administration and site of care, and the evidence needed to assess long-term clinical and economic outcomes. A negative FDA decision, however, would leave those questions unresolved while further clinical and regulatory evaluation of deramiocel continues.

References

1. Cellular, Tissue, and Gene Therapies Advisory Committee July 29, 2026 meeting announcement - UPDATED INFORMATION (as of 7/16/2026). FDA. July 29, 2026. Accessed July 30, 2026. https://www.fda.gov/advisory-committees/advisory-committee-calendar/cellular-tissue-and-gene-therapies-advisory-committee-july-29-2026-meeting-announcement-updated
2. McKenzie H. FDA advisers vote against approval of Capricor’s DMD therapy in chaotic adcomm meeting. BioSpace. July 29, 2026. Accessed July 30, 2026. https://www.biospace.com/fda/fda-advisers-vote-against-approval-of-capricors-dmd-therapy-in-chaotic-adcomm-meeting
3. Capricor Therapeutics provides update on FDA Advisory Committee meeting for deramiocel. News release. Capricor. July 30, 2026. Accessed July 30, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/351/capricor-therapeutics-provides-update-on-fda-advisory
4. Piper Sandler cuts Capricor stock rating after FDA panel vote. Investing.com. July 30, 2026. Accessed July 30, 2026. https://www.investing.com/news/analyst-ratings/piper-sandler-cuts-capricor-stock-rating-after-fda-panel-vote-93CH-4822587
5. The Lancet publishes HOPE-3 data for Capricor Therapeutics’ deramiocel in Duchenne muscular dystrophy. News release. Capricor. July 29, 2026. Accessed July 30, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/350/the-lancet-publishes-hope-3-data-for-capricor