Why was the 2024 label expansion controversial?
Despite EMBARK's failure, in June 2024 Peter Marks again overruled staff — granting traditional approval for ambulatory patients 4+ and accelerated approval for non-ambulatory patients — the group in which the first deaths later occurred.
In June 2024, despite the EMBARK result, the FDA converted Elevidys to traditional approval for ambulatory patients aged 4 and older, and granted accelerated approval for non-ambulatory patients, whose disease is more advanced. Once again, Marks bucked agency staff, relying on his own interpretation of secondary endpoints. ICER's chief medical officer, David Rind, wrote in JAMA that authorising therapies that fail their efficacy endpoints casts doubt on the accelerated approval pathway. The expansion to non-ambulatory patients proved significant because the first deaths later occurred in that group. Supporters argued that DMD is relentlessly fatal and that regulators should accept more uncertainty; critics argued this was the second time a single senior official had overruled reviewers on a Sarepta drug, following Exondys 51 in 2016.