BioCentury
ARTICLE | Clinical News

Exondys 51 eteplirsen regulatory update

September 26, 2016 7:00 AM UTC

FDA granted accelerated approval to Exondys 51 eteplirsen from Sarepta to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Sarepta will price the drug at an expected net annual cost of $300,000 for a 25 kg boy, taking into account changes in weight-based dosing over time and expected compliance rates. A 2 mL vial of the drug will cost $1,600, and a 10 mL vial will cost $8,000. The approved dose is 30 mg/kg once weekly. Exondys 51 is phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA.

The approval is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some treated patients. In April, FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7-6 that Sarepta had not shown substantial evidence that eteplirsen induced dystrophin expression “to a level that is reasonably likely to predict clinical benefit” (see BioCentury, May 2). ...