An FDA advisory committee voted 9-3 against Capricor Therapeutics’ cell therapy deramiocel for Duchenne muscular dystrophy-associated cardiomyopathy after concluding the available evidence did not demonstrate the therapy’s effectiveness.
The vote followed FDA briefing documents released ahead of the meeting that concluded the phase 3 HOPE-3 trial failed to meet its prespecified primary efficacy endpoint and found no statistically significant difference between deramiocel and placebo on a key secondary endpoint measuring left ventricular ejection fraction. Agency reviewers also said they were unable to identify a patient subgroup that may benefit from the therapy.
During the meeting, Capricor argued the HOPE-3 trial demonstrated a treatment benefit using a revised statistical analysis plan and pointed to data published in The Lancet. FDA reviewers maintained the trial’s efficacy findings were not supported under the prespecified analyses, and several committee members said the results were too dependent on the statistical methods used.
Although panelists said the therapy’s upper limb function data warranted additional study, they concluded the evidence did not support efficacy for the proposed indication. FDA advisory committee recommendations are nonbinding, and the agency is expected to decide on Capricor’s biologics license application by Aug. 22.
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