What is the case for and against Elevidys?
Supporters cite a fatal disease with no alternatives and biological activity on secondary measures; critics — including a Health Affairs analysis titled "Unsafe, Not Effective, $3.2 Million Per Dose" — cite two primary-endpoint failures, two regulatory overrides and deaths linked to treatment.
Supporters, including many DMD families and clinicians, argue that without treatment boys with DMD lose the ability to walk and die young, that micro-dystrophin expression shows the therapy is biologically active, and that secondary measures suggest it modifies the disease course. They argue regulators must accept more uncertainty for fatal rare diseases. Critics, including a Health Affairs analysis titled "Unsafe, Not Effective, $3.2 Million Per Dose", argue that it failed its primary endpoint twice, was approved by one official overriding review teams, has caused deaths, and is priced at a level that strains payers without proven functional benefit. They argue accelerated approval only works when surrogate endpoints reliably predict outcomes that matter to patients. Longer-term follow-up data will be decisive.