Duchenne Muscular Dystrophy
ICER will assess the comparative clinical effectiveness and value of treatments for Duchenne muscular dystrophy. This evidence review will examine two exon-skipping therapies: eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta). Eteplirsen was approved by the FDA in September 2016 for patients with a confirmed mutation in the dystrophin gene that is amenable to exon 51 skipping. Sarepta has announced its intention to file for accelerated approval of golodirsen by the end of 2018. This list is tentative and subject to change.
The New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.