Why is Sarepta so controversial?
Twice, senior FDA officials overruled their own review teams to approve Sarepta drugs on surrogate biomarkers without confirmed functional benefit — then in 2025 patient deaths from acute liver failure led to a public standoff over whether to halt shipments.
Sarepta sits at the centre of a long-running argument about how much evidence regulators should demand before approving drugs for devastating rare diseases. Twice, senior FDA officials have overruled the agency's own reviewers to approve Sarepta products on the basis of a biomarker (dystrophin or micro-dystrophin production) rather than demonstrated improvement in how patients function. Patient families and advocacy groups have strongly supported these approvals, arguing that boys with DMD cannot wait.
Critics, including former FDA staff and pricing experts, argue Sarepta has benefited from lowered standards and charged very high prices for drugs whose benefit remains unproven. The debate intensified in 2025 when deaths from acute liver failure were reported after treatment with Elevidys and Sarepta initially resisted an FDA request to halt shipments. Sarepta maintains its therapies offer meaningful benefit and that it has acted responsibly on safety.