Capricor Therapeutics saw its stock tumble by 12% after the U.S. Food and Drug Administration (FDA) announced it will convene an expert advisory panel to review the company’s lead cell therapy, deramiocel, for Duchenne muscular dystrophy (DMD)-related cardiomyopathy.
The move introduces a new layer of scrutiny and potential uncertainty for Capricor’s high-profile therapy, which is seeking to become the first approved treatment for this life-threatening heart complication in DMD patients.
FDA to Convene Advisory Committee
The FDA’s decision to assemble an external panel of experts comes ahead of its scheduled action date of August 31, 2025, for Capricor’s Biologics License Application (BLA) for deramiocel.
While the agency has not identified any significant deficiencies in its ongoing review, the advisory committee will provide independent recommendations on the therapy’s safety and efficacy before a final regulatory decision is made.
The date for the advisory panel meeting has not yet been announced, but it is expected to take place prior to the PDUFA deadline.
Market Reaction and Analyst Perspective
Following the FDA’s announcement, Capricor shares dropped nearly 15% in early trading, reflecting investor concerns about potential delays or additional hurdles in the approval process.
However, some analysts argue the decline may be overdone, noting that the opportunity to present data before an expert panel could ultimately strengthen the case for deramiocel. The company remains optimistic, emphasizing that no major review issues have been flagged by the FDA to date.
Background: Deramiocel’s Promise for DMD Cardiomyopathy
Duchenne muscular dystrophy is a rare genetic disorder marked by progressive muscle weakness, including the heart muscle, which often leads to fatal cardiomyopathy.
Deramiocel, Capricor’s investigational cell therapy, has shown promise in clinical trials by attenuating heart muscle deterioration in DMD patients-a group with limited treatment options and high unmet medical need.
The FDA has granted deramiocel Priority Review status, with its BLA supported by data from Phase 2 HOPE-2 and extension studies. If approved, deramiocel would become the first therapy specifically targeting DMD-related cardiomyopathy, potentially transforming care for thousands of patients in the U.S.