FDA Advisory Committee Evaluates Muscular Dystrophy Treatment
The Spin
Narrative A
The FDA's own briefing makes clear that Capricor's HOPE-3 trial failed to meet its pre-specified primary and secondary endpoints, and that the company then altered its statistical analysis plan after unblinding, a move that undermines the entire evidentiary foundation. Approving a therapy on this record would set a dangerous precedent for rare disease drug development.
Narrative B
Capricor's HOPE-3 study definitively shows that it met its primary skeletal muscle endpoint and key cardiac endpoint, with deramiocel slowing upper-limb decline by 54% and preserving heart function. The publication of these results in The Lancet, with its rigorous independent peer review, is telling and further validates the trial's design, methodology and findings.
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