July 29 (Reuters) – The U.S. FDA’s panel of independent advisers on Wednesday voted against the benefits of Capricor Therapeutics’ cell therapy, deramiocel, for treating cardiomyopathy in Duchenne muscular dystrophy, a rare muscle-wasting disease.
The panel voted 9 to 3 against deramiocel. The recommendation is a setback for Capricor as it seeks approval for what could become the first treatment specifically targeting heart disease in Duchenne patients.
The vote is in line with FDA staff concerns that Capricor had not provided sufficient evidence of effectiveness of deramiocel, while questioning changes to key analyses after the company’s late-stage study was completed.
Earlier this week, FDA reviewers also questioned whether patients enrolled in the trial had Duchenne-related cardiomyopathy, saying that heart-pumping function was normal on average at the start of the study.
“We are completely shocked at how they decided to review and analyze this data. It just shows that you can take any clinical-trial data and make it look quite dark and cloudy when you apply the wrong statistical analysis plan,” Capricor CEO Linda Marbán said on Monday.
Last year, the Food and Drug Administration had declined to approve the therapy and sought additional data after saying the treatment did not meet efficacy requirements.
(Reporting by Sahil Pandey and Christy Santhosh in Bengaluru; Editing by Sahal Muhammed)
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