An FDA advisory committee is scheduled to review Capricor Therapeutics’ cell therapy deramiocel after agency reviewers concluded the available clinical data do not provide substantial evidence that the treatment is effective for Duchenne muscular dystrophy.
FDA reviewers said the phase 3 HOPE-3 study did not meet its prespecified primary efficacy endpoint, and no statistically significant difference was observed between deramiocel and placebo on a key secondary endpoint measuring left ventricular ejection fraction, according to FDA briefing documents uploaded ahead of the July 29 meeting. The agency also said it was unable to identify a patient subgroup that may benefit from the therapy after reviewing data from HOPE-3 and the earlier HOPE-2 study.
The FDA said the original biologics license application received a complete response letter because of a lack of evidence of effectiveness. Agency reviewers also said the current benefit-risk assessment appears unfavorable because the submitted data do not provide substantial evidence of effectiveness and the therapy carries observed safety risks, including hypersensitivity reactions and anaphylaxis.
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