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FDA Panel Rejects Heart Therapy for Duchenne Patients
30 Jul
Summary
- Advisers voted against Capricor's cell therapy for Duchenne cardiomyopathy.
- The panel's recommendation is a setback for the first targeted heart disease treatment.
- FDA staff questioned the therapy's effectiveness and study analysis changes.
An independent advisory panel for the U.S. Food and Drug Administration (FDA) has voted against the therapeutic benefits of Capricor Therapeutics' cell therapy, deramiocel. The therapy was intended for patients with cardiomyopathy associated with Duchenne muscular dystrophy.
The panel's 9-to-3 recommendation marks a significant setback for Capricor, which had aimed to secure approval for what could have been the inaugural treatment specifically addressing heart disease in Duchenne patients.
This decision aligns with concerns previously voiced by FDA staff regarding the evidence of deramiocel's effectiveness. Additionally, questions were raised about alterations to key analytical methods post-completion of the company's late-stage study.
Earlier in the week, FDA reviewers also expressed doubt about whether the trial participants indeed had Duchenne-related cardiomyopathy, noting that heart-pumping function was generally normal at the study's outset. The FDA had previously declined to approve the therapy last year, requesting further data after determining it did not meet efficacy requirements.
