What are the main criticisms of Sarepta's approach?
Four points: approvals resting on surrogate biomarkers the FDA's own scientists doubted; prices of $300,000/year and $3.2 million/dose for unconfirmed benefit; slow or negative confirmatory trials; and safety disclosures in 2025 that fell short of expected transparency.
Critics make four points. First, that its key approvals rested on surrogate biomarkers the FDA's own scientists doubted, and on the personal judgement of senior officials overriding review teams. Second, that its prices, around $300,000 a year for Exondys 51 and $3.2 million per dose for Elevidys, are extraordinarily high for unconfirmed benefit; ICER's chief medical officer argued in JAMA that such approvals cast doubt on the accelerated approval pathway. Third, that confirmatory trials have been slow or negative without leading to withdrawal.
Fourth, that Sarepta's handling of safety disclosures in 2025 fell short of expected transparency. Sarepta's defenders, including many families, counter that DMD is fatal, that regulators must accept more uncertainty in rare diseases, and that the company has brought the first treatments to a community that had none.