PDUFA Date

Capricor's deramiocel faces Aug. 22 FDA decision after CRL reversal

The FDA lifted a 2025 Complete Response Letter and resumed review of deramiocel for DMD cardiomyopathy, setting up an August 22 decision built on HOPE-3 data showing the trial hit its primary endpoint.

The FDA has a PDUFA target action date of August 22, 2026, for Capricor Therapeutics' resubmitted BLA for deramiocel in Duchenne muscular dystrophy cardiomyopathy, after lifting a Complete Response Letter issued in July 2025.

By AppliedXLJuly 22, 2026Trial NCT05126758

Executive Summary

Capricor Therapeutics faces an FDA action date in about four weeks on a Biologics License Application the agency previously rejected once and is now reviewing on resubmission.
The application rests on a completed Phase 3 trial that the sponsor has said met its primary endpoint and a key secondary cardiac endpoint, though the agency's independent read of that same data drove the prior rejection.
An approval would create the first therapy that the sponsor and its designations indicate directly addresses the cardiac, and not only skeletal, muscle deterioration that drives mortality in this disease population.
The agency's Orphan Drug and Regenerative Medicine Advanced Therapy designations signal rare-disease unmet need and cell-therapy development support, not a forecast of the coming decision.

The catalyst

The FDA resumed review of Capricor Therapeutics' BLA for deramiocel in DMD cardiomyopathy after lifting the Complete Response Letter it issued in July 2025, and assigned a PDUFA target action date of August 22, 2026. The submission is classified as a Class 2 resubmission, and the company said the FDA had not identified any potential review issues in its response as of the March 2026 disclosure. Capricor Chief Executive Linda Marban said the company is "encouraged by the FDA's acknowledgment of our response to the Complete Response Letter and its continued review of our BLA for Deramiocel". Capricor also said it expects to be eligible for a Priority Review Voucher if the application is approved. Capricor

Probability of Success

Endpoint Met91%

Completes79%

Clinical Significance28%

Regulatory84%

The trial behind the filing

The BLA is supported by the Phase 3 HOPE-3 trial (NCT05126758), a randomized, quadruple-masked, placebo-controlled study that enrolled 106 patients with DMD and is now Active, not recruiting. The trial tested change in upper-limb function as its primary endpoint, with left ventricular ejection fraction among its 22 secondary endpoints. Capricor has said HOPE-3 met its primary endpoint on the Performance of Upper Limb (PUL 2.0) measure (p=0.03) and its key secondary cardiac endpoint on LVEF (p=0.04), with a favorable safety profile, and presented these results at the American Academy of Neurology meeting on April 21, 2026. The trial's primary completion date moved three times over the study's course, most recently landing at June 18, 2025. NCT05126758+1

Long-term extension data

Capricor also presented five-year data from the open-label extension of its earlier HOPE-2 trial (NCT04428476) on June 27, 2026, showing that the nine patients remaining in that cohort had a mean PUL 2.0 decline of less than 5 points over five years and stable LVEF, with no new safety signals. That readout does not change what the FDA is reviewing in the current BLA cycle, but it extends the durability picture behind deramiocel's mechanism, since HOPE-2 is the earlier-phase precursor to the pivotal HOPE-3 dataset now under review. NCT05126758

The regulatory posture

Deramiocel holds Orphan Drug Designation from both the FDA and the European Medicines Agency, and Regenerative Medicine Advanced Therapy designation in the United States, both associated with the DMD indication. These designations signal that the FDA has recognized DMD as a rare disease with unmet need and has offered the cell-therapy program development support; they are not a signal of the odds of the pending decision. No FDA-approved therapy in DMD carries a labeled indication for cardiomyopathy specifically: the drugs approved for DMD, including exon-skipping oligonucleotides such as viltolarsen and golodirsen and the gene therapy delandistrogene moxeparvovec, target dystrophin production in skeletal muscle rather than cardiac tissue directly. Capricor

The competitive field

The DMD treatment landscape is mechanistically diverse and mature on the skeletal-muscle side, spanning exon-skipping oligonucleotides, a gene therapy already approved for ambulatory patients, an HDAC inhibitor, and glucocorticoid-class agents. Deramiocel's modality, allogeneic cardiosphere-derived cell therapy, has no approved precedent in DMD and only sparse same-modality activity in the indication: the nearest parallel is Solizmestrocel, a Phase 2 cell therapy from MED Institute in DMD, and the nearest modality precedent outside the indication is Brainstorm-Cell Therapeutics' debamestrocel, a Phase 3 cell therapy in ALS. None of the active DMD competitors share deramiocel's mechanism of targeting cardiac muscle preservation through immunomodulatory cardiosphere-derived cells, so the coming decision turns on the FDA's read of the deramiocel data package itself rather than on a comparative efficacy bar set by a rival cardiac therapy.

This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.