FDA and Capricor clash over Duchenne cell therapy efficacy at advisory committee

At an FDA advisory committee meeting, the agency and Capricor Therapeutics presented conflicting views on the efficacy data for the company's experimental cell therapy aimed at Duchenne muscular dystrophy.

The FDA panel members questioned the robustness of the trial results, citing concerns about the magnitude of benefit and variability among patients.

Capricor defended the data, arguing that the therapy showed meaningful improvement in muscle function and meets the unmet need for this fatal disease.

The disagreement highlighted the regulatory hurdles the therapy faces before possible approval, and investors will be watching the outcome closely.