UPDATED: Sarepta's Exondys 51 is not cost-effective, nor particularly beneficial for DMD patients — ICER

Parents with children suffering from Duchenne muscular dystrophy (DMD) — a rare, progressive muscle wasting disease that disproportionately affects young boys — cheered in 2016 when Sarepta Therapeutics’ Exondys 51 (eteplirsen) was controversially approved by the FDA, under pressure from patient advocates and despite stiff opposition from within and outside the agency. Critics lambasted the agency’s decision, citing insufficient data — and now, an increasingly influential ICER has concurred: The underlying evidence supporting the drug is sparse, and given its current pricing, it is certainly not cost-effective.