The FDA advisory panel voted 9-3 against Capricor's cell therapy deramiocel for cardiomyopathy in Duchenne muscular dystrophy, citing insufficient evidence of benefit and concerns over post-hoc changes to key analyses. Reviewers also questioned whether trial participants truly had Duchenne-related cardiomyopathy, since average cardiac pumping function was normal at baseline. Capricor's CEO strongly criticized the review process; the FDA had previously declined approval and asked for more data.
FDA Advisory Panel Rejects Capricor’s Duchenne Heart Therapy in 9-3 Vote
Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly
July 29 (Reuters) - An independent advisory panel to the U.S. Food and Drug Administration voted 9-3 on Wednesday that Capricor Therapeutics' cell therapy, deramiocel, has not demonstrated clear benefits for treating cardiomyopathy associated with Duchenne muscular dystrophy, a progressive, rare muscle-wasting disease.
The recommendation marks a significant setback for Capricor as it pursues approval of what could have been the first therapy aimed specifically at Duchenne-related heart disease.
Panel Concerns
FDA staff and panel members said the company did not provide sufficient evidence of deramiocel's effectiveness. Reviewers also raised concerns about changes made to key analyses after the late-stage clinical trial was completed, calling those post-hoc revisions into question.
Earlier in the week, FDA reviewers additionally questioned whether many trial participants actually had Duchenne-related cardiomyopathy, noting that average measures of cardiac pumping function (ejection fraction) were within normal ranges at baseline.
"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,"
The FDA previously declined to approve deramiocel last year and requested additional data after concluding the treatment had not met efficacy requirements.
What Happens Next
While the advisory panel's recommendation is influential, it is not binding. The FDA will consider the panel's advice alongside its own reviewers' findings before making a final decision. Capricor may provide further data, pursue additional analyses, or seek discussions with regulators to address the concerns raised.
(Reporting by Sahil Pandey and Christy Santhosh in Bengaluru; Editing by Sahal Muhammed)
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