FDA Panel Votes 9-3 Against Capricor Deramiocel for DMD

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 against Capricor Therapeutics (CAPR) on July 29, 2026, concluding that the HOPE-3 Phase 3 trial did not provide substantial evidence that deramiocel is effective for treating cardiomyopathy in Duchenne muscular dystrophy.

The vote sends Capricor’s lead asset toward a likely rejection at its August 22 PDUFA date. CAPR closed at $3.85 on July 31 (Polygon), down 80.6% from $19.83 after FDA briefing documents published July 27 disputed the company’s efficacy claims. The stock had been halted during the meeting. Market cap has fallen below $100 million.

Update August 27, 2026: CAPR closed at $9.36 on August 26 (Polygon), up 36.8% from the $6.84 August 20 close. Oppenheimer upgraded the stock on August 25 following the FDA’s confirmation that deramiocel’s PDUFA is extended to November 22 (upper-limb-only claim).

Update August 21, 2026: The August 22 PDUFA is one day away (August 22 is a Saturday; the FDA may act Friday August 21 or Monday August 24). Two developments have reshaped the setup. First, on August 13 alongside Q2 results, Capricor outlined a “clearer regulatory path” for deramiocel — widely read as a potential pivot to an upper-limb (skeletal) indication rather than the cardiomyopathy claim the committee rejected — and CAPR surged 67% after-hours. Second, on August 20, STAT’s Adam Feuerstein reported the FDA is “heading for” rejecting the therapy, calling Capricor’s submission of open-label extension data “a stall tactic” and arguing only a new randomized trial could secure approval. CAPR closed at $6.84 on August 20 (Polygon), up 77% from the $3.85 July 31 close but still down 66% from the $19.83 pre-briefing reference.

Update August 22, 2026: On August 21, Capricor confirmed it will submit a BLA amendment narrowing deramiocel’s proposed indication to upper-limb dysfunction only — and the FDA is expected to extend the August 22 PDUFA once it receives the amendment. See our amendment coverage.

Update August 25, 2026: The FDA confirmed on August 24 that it extended deramiocel’s PDUFA to November 22, 2026, classifying the upper-limb-only amendment as a major (three-month) extension. No approval-or-CRL decision lands until then. See our amendment coverage.

What the committee said

The sole voting question asked whether HOPE-3 provides substantial evidence of effectiveness for deramiocel in DMD cardiomyopathy. Nine of twelve voting members said no.

Steven Pavlakis, MD, of SUNY Downstate Health Sciences University, called the data “very fragile.” John Teerlink, MD, of UC San Francisco, acknowledged “some glimpses there” but said he could not “consider it strong support” at this time. Both voted no.

The meeting centered on a dispute over the statistical analysis plan. FDA reviewer Prateek Shukla, MD, presented the agency’s analysis using SAP version 1.1, which the FDA said was prespecified. That analysis showed a mean difference of just 0.66 points on the Performance of Upper Limb 2.0 scale (95% CI -0.45 to 1.77, P=0.24) between deramiocel and placebo at 12 months. The key secondary endpoint, left ventricular ejection fraction, showed a change of -0.04% (P=0.97).

Capricor’s analysis used a later SAP version (3.0, finalized before unblinding, the company says) and produced a 4.55-point PUL 2.0 difference (P=0.029). CEO Linda Marban compared the FDA’s approach to “your professor grading your term paper on an early draft you had never even submitted.”

Why the cardiac indication mattered

The committee focused on cardiomyopathy because that was the indication Capricor sought in its BLA, even though HOPE-3’s primary endpoint measured upper limb function. Karim Mikhail, acting director of the FDA’s Center for Biologics Evaluation and Research, said the meeting was framed around the cardiac claim specifically.

Duchenne muscular dystrophy causes progressive degeneration of skeletal and cardiac muscle. Cardiomyopathy is the leading cause of death in DMD patients. If approved, deramiocel would have been the first cell therapy for DMD cardiomyopathy.

What to watch next

The August 22 PDUFA has passed without a decision — the FDA extended the review to November 22, 2026 after classifying Capricor’s upper-limb-only amendment as a major amendment (confirmed August 24). The FDA is not bound by advisory committee votes but follows them in most cases. A 9-3 negative vote, combined with the agency’s own briefing documents concluding the trial failed its endpoints and STAT’s August 20 report that the FDA is “heading for” rejection, still leans against approval. The key question is whether Capricor’s pivot to an upper-limb (skeletal) indication — which drove the 67% after-hours surge on August 13 — changes the calculus, but the vote and the briefing documents both centered on the cardiac claim, and STAT characterizes the company’s open-label extension submission as “a stall tactic.” See our amendment coverage for the November 22 setup.

Capricor’s options are narrow. The company could request a Type A meeting with the FDA to discuss paths forward, resubmit with additional analyses, or conduct another trial. The Lancet published the HOPE-3 results the same day as the vote, but the published analysis reflects the company’s SAP, not the FDA’s.

For background on how this BLA reached the AdComm, including the rescinded Complete Response Letter from 2025, see our AdComm preview.

Endpoints News coverage | FDA AdComm meeting announcement | HOPE-3 on ClinicalTrials.gov