By Christy Santhosh and Sahil Pandey
July 29 (Reuters) – The U.S. FDA’s panel of independent advisers on Wednesday voted against effectiveness data of Capricor Therapeutics’ cell therapy, deramiocel, for a heart condition related to Duchenne muscular dystrophy.
The nine-to-three vote aligns with FDA staff concerns that the company had not provided sufficient evidence of effectiveness of deramiocel, while questioning changes to key analyses after the company’s late-stage study was completed.
The FDA staff had said Capricor changed how it measured the main outcomes, shifting arm-function analysis from scores on a 42-point test to percentage changes and changing heart-function measurement from changes in blood pumped per beat to a ranking of patients’ results, according to briefing documents published on Monday.
Capricor did not immediately respond to Reuters’ request for comment on Wednesday.
Cynthia Tifft, a pediatrician and gene-therapy expert at the National Institutes of Health (NIH) who serves on the advisory panel, said she voted against the therapy because the evidence on its heart-related benefits was not compelling.
“It pains me” to vote no, Tifft said, adding that a larger and longer trial using different end goals might have been better positioned to show whether the treatment helped preserve arm and hand function.
FDA reviewers had 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.
Lori Dodd, panel member and a senior biostatistician at the NIH, said the company’s cardiac evidence was weakened by missing data and uncertainty over whether its main measurement, which indicates how well the heart pumps, could stand in for a meaningful patient benefit.
She described the results as “quite fragile” and said more sensitivity analyses were needed to test whether the findings held under different assumptions.
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.
While the FDA is not required to follow the advice of its outside experts, it often considers their recommendations when making approval decisions. The agency is expected to decide on the therapy by August 22.
Capricor shares have fallen more than 66% since the briefing documents with the FDA staff’s concerns were published on Monday.
(Reporting by Sahil Pandey and Christy Santhosh in Bengaluru; Editing by Sahal Muhammed and Sriraj Kalluvila)
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