How was Elevidys first approved?
Accelerated approval in June 2023 for ambulatory boys aged 4–5 on an 8-6 advisory vote — with the FDA's clinical, pharmacology and statistics teams finding the data inadequate — because the biologics centre head overruled his review staff.
The FDA granted accelerated approval on June 22, 2023, for ambulatory boys aged 4 and 5. The basis was micro-dystrophin expression, which the FDA judged reasonably likely to predict benefit, rather than proven improvements in function. An FDA advisory committee had voted only 8-6 in favour. The FDA's own reviewers were more sceptical: briefing documents said micro-dystrophin levels only showed the gene was being expressed, rather than providing insight into a pharmacologic effect on the disease. According to a Health Affairs analysis, the clinical, pharmacology and statistics teams found the data not adequate to meet the threshold for approval. Peter Marks, head of the FDA's biologics centre, decided against the review team's recommendation and approved it.