Key Takeaways
- Capricor Therapeutics responded to FDA’s rejection of its Duchenne muscular dystrophy therapy, deramiocel, seeking clarity on concerns outlined in the Complete Response Letter.
- The FDA’s new policy to publicly share rejection letters coincided with this announcement, highlighting transparency in regulatory processes.
- Despite the setback, Capricor remains optimistic about obtaining approval for deramiocel, with plans for a BLA resubmission informed by upcoming clinical trial data.
FDA Rejection and Capricor’s Response
Capricor Therapeutics has publicly addressed the FDA’s rejection of its Duchenne muscular dystrophy (DMD) cell therapy, deramiocel, following the release of new Complete Response Letters (CRLs) by the FDA on September 4. This situation arose from the agency’s unprecedented move to publish rejection letters simultaneously with issuing them to companies, a significant shift in FDA policy.
Capricor’s rejection notice was issued on July 9, prompting the company to respond on July 16 to clarify FDA concerns. The biotechnology firm expressed discontent regarding the lack of prior notification about the public revelation of the rejection. CEO Linda Marbán, Ph.D., emphasized the importance of transparency, especially concerning therapies that could significantly impact patients with severe conditions like DMD.
The FDA’s reasons for denying deramiocel centered on identified deficiencies in Capricor’s Phase 2 DMD trial. According to the CRL, the HOPE-2 trial did not meet its primary efficacy endpoint, which was a change in neuromuscular function. However, Capricor contended that the endpoint was indeed met using what it described as a more suitable statistical analysis technique, yielding a p-value of 0.014.
Further complicating Capricor’s application was the FDA’s stance on the data supporting the treatment of DMD-associated cardiomyopathy. The agency highlighted that the evidence included 50 secondary and exploratory endpoints that had not been pre-specified for hypothesis testing, raising the potential for false positives. Capricor rebutted this assertion, arguing that discussions with the FDA prior to submitting its Biologics License Application (BLA) indicated that all endpoints were deemed pre-specified.
Capricor expressed confusion over the shift in feedback from the FDA, noting that its clinical plans had been previously agreed upon with the agency. The biotechnology company is vying for approval, eagerly awaiting the official minutes from a recent Type A meeting held with the FDA to understand the next steps better.
Despite the denial, Capricor remains hopeful for deramiocel’s future approval. CEO Marbán shared plans for a BLA resubmission bolstered by expected topline data from the upcoming HOPE-3 trial, anticipated in the fourth quarter of 2025.
As the FDA continues to scrutinize companies seeking approvals for promising cell and gene therapies under the leadership of Dr. Vinay Prasad, Capricor’s persistence indicates resilience in the face of regulatory challenges. This ongoing narrative reflects broader trends in the biotech field as the industry grapples with rigorous standards and evolving regulatory practices.
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