The FDA has denied Capricor Therapeutics’ request for clearance of a Duchenne muscular dystrophy (DMD) cell treatment, prompting questions on the potential reversal of the previous administration’s leniency under the agency’s new management. Capricor’s stock price declined by nearly 40% to $7.11 in premarket trading.
FDA regulators determined that the deramiocel proposal did not meet the criteria for significant proof of efficacy and requested further clinical data, according to the biotech company. Capricor’s convoluted path to its first FDA submission suggests that the firm may swiftly fulfil the need for more clinical evidence.
In 2022, Capricor initiated a phase 3 study after a consultation with the FDA over the next stages for the program. At that time, the biotechnology firm chose not to pursue approval based on a phase 2 database that was smaller than anticipated. Capricor reevaluated its approach after three years of information from an open-label expansion of the phase 2 study, which demonstrated persistent improvements in cardiac function, evidence that validated the recently rejected application.
Capricor anticipates obtaining results from a double-blind, placebo-controlled phase 3 study including 104 participants in the third quarter. Capricor CEO Linda Marbán said that the business is confident that the phase 3 findings, together with current evidence, may address the concerns highlighted by the FDA about the treatment of cardiomyopathy linked to DMD.
Marbán said that Capricor was taken aback by the complete response letter (CRL), noting that the business adhered to the FDA’s recommendations throughout the whole process. The recent restructure and layoffs at the FDA indicate that the individuals who offered advice and evaluated the submission might be different from those writing the CRL.
Capricor completed its application for FDA clearance of deramiocel for DMD cardiomyopathy in January. At that time, Dr. Nicole Verdun was overseeing the cell and gene therapy office, while Dr. Peter Marks served as the head of the Centre for Biologics Evaluation and Research. Neither of them remains in their respective spots.
During an earnings call a few months back, Marbán said that Verdun had been engaged with the deramiocel project since the start of 2024. Five weeks later, the head of cell and gene therapy was allegedly placed on administrative leave and removed from the agency. Days after Verdun’s leave, the FDA abandoned preparations to convene an advisory group regarding the deramiocel proposal.
Capricor said that the CRL also identifies deficiencies in the chemistry, production, and controls section of the submission. The biotech asserts that it has resolved the majority of the lingering issues in previous conversations with the FDA; but, the timing of the CRL precluded their assessment by the agency. The FDA sent the CRL seven weeks prior to the August 31 PDUFA deadline for the submission.
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