Did Elevidys pass its confirmatory trial?
No — the phase 3 EMBARK trial failed its primary motor-function endpoint (2.6 vs 1.9 points, not statistically significant), yet Sarepta's CEO called it evidence of effectiveness and the FDA later expanded the label anyway.
No. The phase 3 EMBARK trial enrolled 125 ambulatory boys aged 4 to 7 and measured change in the North Star Ambulatory Assessment (NSAA) motor score at 52 weeks. Treated boys improved by 2.6 points versus 1.9 points on placebo, a difference that was not statistically significant, so the trial failed its primary endpoint. Some functional measures, such as time to rise from the floor, favoured treatment, but those were not statistically significant either. Micro-dystrophin expression averaged 34.29% versus zero on placebo. Sarepta's CEO said the study "met the standard" to show evidence of effectiveness and that the drug "alters" the disease course. Critics noted the drug had now fallen short of its primary endpoint in two studies.