Biotech company Capricor Therapeutics (Nasdaq: CAPR) faces heightened regulatory uncertainty after US Food and Drug Administration (FDA) reviewers questioned whether its resubmitted Biologics License Application (BLA) for deramiocel provides substantial evidence of effectiveness in cardiomyopathy in male patients with Duchenne muscular dystrophy.
FDA briefing documents, released ahead of Wednesday’s meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee, said the pivotal Phase III HOPE-3 study did not meet its pre-specified primary and secondary efficacy endpoints under the original statistical analysis plan. Reviewers also raised concerns about later changes to endpoint definitions, analytical methods and handling of missing or intercurrent data.
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