The FDA questioned the efficacy of Capricor’s deramiocel in Duchenne muscular dystrophy ahead of an advisory committee meeting. A review team said it was unable to identify enough evidence that the Phase 3 objectives were met, contradicting the company’s earlier claims after the sponsor reported meeting primary and secondary endpoints. Capricor responded by arguing the FDA’s analysis relied on an incorrect or outdated statistical analysis plan, while the company said its positive analysis was based on a finalized plan prior to unblinding. The dispute sets up a high-stakes adcom “cage match” over how to interpret the trial’s statistical and clinical data. The situation highlights how tightly regulator conclusions can depend on analysis plan integrity for gene and cell therapy approvals, even when companies report endpoint success.