BioCentury
ARTICLE | Regulation

Digging into dosing

How Sarepta, FDA can determine dose for Exondys 51's confirmatory trial

September 26, 2016 7:00 AM UTC

Clinical and preclinical data from Sarepta Therapeutics Inc. give hope that higher dosing of Exondys 51 eteplirsen could result in substantially higher dystrophin production than seen in its clinical trials. Figuring out how much and how often to give Exondys 51 will require a combination of extrapolation from preclinical data and guesswork, because the company never established a robust dose response relationship in the clinic.

Sarepta's clinical program tested doses of Exondys ranging from 0.5 mg/kg/week to 50 mg/kg/week. The drug is a phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA...