FDA Extends Deramiocel PDUFA Date to November 2026 After Capricor Submits Refined DMD Indication

Capricor Therapeutics announced that the FDA has extended the PDUFA target action date for its BLA for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026.¹

Why was the amendment submitted?

The amendment follows the July 2026 FDA advisory committee meeting, where agency reviewers disputed Capricor's claim that HOPE-3 met its pre-specified primary and secondary efficacy endpoints, a disagreement centered on which version of the trial's statistical analysis plan should govern the results.² BioPharm International^© covered that dispute in detail, including Capricor's response defending SAP version 3.0 as the analysis plan finalized before unblinding; see our earlier coverage of the FDA-Capricor efficacy dispute for the full background. The amendment adds long-term follow-up data on that same endpoint rather than introducing a new one, giving the agency an extended dataset on the outcome measure already at the center of the disagreement.

What did the FDA accept for review?

The amendment includes 24-month open-label extension data from HOPE-3 along with additional robustness analyses.¹ The FDA's Center for Biologics Evaluation and Research accepted the amendment for review, citing the significant unmet medical need in DMD, and classified the submission as a major amendment, extending the PDUFA date by three months to allow additional review time.¹

"With an additional year of follow-up from HOPE-3, we now have one of the most extensive clinical datasets evaluating upper limb function in Duchenne," said Linda Marbán, PhD, chief executive officer of Capricor Therapeutics. "HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, and we believe the additional open-label data and further analyses included in the amendment strengthen the evidence supporting a refined proposed indication."¹

Why does upper limb function matter in DMD?

DMD is a progressive, X-linked genetic disorder affecting an estimated 15,000 individuals in the US, caused by the absence of functional dystrophin, a protein essential to muscle cell structure.¹ Cardiomyopathy from deteriorating heart muscle is the disease's leading cause of death, and there is currently no cure.¹ Marbán pointed to patient and clinician testimony from the July advisory committee meeting underscoring the role upper limb function plays in patient independence. "In a progressive disease where function, once lost, cannot be recovered, we believe preservation of upper limb function has the potential to translate into meaningful differences in patients' independence and daily lives," she said.¹

How does deramiocel work?

Deramiocel consists of allogeneic cardiosphere-derived cells that Capricor says exert immunomodulatory and anti-fibrotic effects supporting preservation of skeletal and cardiac muscle function in DMD.¹ The cells act by secreting exosomes that target macrophages, shifting their expression profile toward a healing rather than pro-inflammatory phenotype.¹ Deramiocel holds Orphan Drug, RMAT, and Rare Pediatric Disease designations in the US, and Orphan Drug and ATMP designations in Europe; the Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval.¹

What's next?

The FDA's review is now set to conclude by the new PDUFA target action date of November 22, 2026.¹ The FDA's decision will determine whether deramiocel, currently Capricor's lead candidate, receives approval for DMD-associated cardiomyopathy under this refined indication.

References

1. Capricor Therapeutics. Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA. Press release. Published August 24, 2026. Accessed August 24, 2026.
2. Schoenthaler, E. FDA Disputes Capricor's Phase 3 Efficacy Claims for Duchenne Cell Therapy Deramiocel. BioPharm International. Published July 28, 2026. Accessed August 24, 2026.