How was the Elevidys gene therapy approved?
An advisory committee voted only 8-6 in favour, FDA clinical and statistics teams found the data inadequate, and the biologics centre head again overruled staff — then expanded the label in 2024 even after the confirmatory trial failed its primary endpoint.
Elevidys, a one-time gene therapy priced at $3.2 million, received accelerated approval in June 2023 for ambulatory boys aged 4 and 5. An advisory committee voted only narrowly in favour, 8-6. The FDA's clinical, pharmacology and statistics review teams had found the data not adequate to meet the threshold for approval, and it was Peter Marks, head of the FDA's biologics centre, who decided against their recommendation.
In October 2023 the confirmatory EMBARK trial failed its primary endpoint on motor function. Even so, in June 2024 Marks again bucked agency staff, granting traditional approval for ambulatory patients aged 4 and over and accelerated approval for non-ambulatory patients. Sarepta argued secondary endpoints showed meaningful benefit.