Patients Can't Rescue Sarepta's Eteplirsen
This article was originally published in The Pink Sheet Daily
Hours of testimony was clearly compelling, but advisory committee agreed with FDA concerns about the Duchenne muscular dystrophy drug's efficacy.
You may also be interested in...
As with many rare diseases, a clear natural history would make deciphering the data easier, but for now what Sarepta characterizes as stabilization is seen by FDA reviewers as expected improvement based on previous measures of disease progression at younger ages.
Only four of 14 voting panelists are regular members of the Cellular, Tissues and Gene Therapies Advisory Committee, with scheduling conflicts precluding participation by many others, the FDA said. Three of the temporary members are veterans of prior muscular dystrophy drug reviews.
We look back at another of the greatest FDA advisory committees of the last 20 years. This time: the nearly 12-hour meeting in 2016 on Sarepta’s eteplirsen for Duchenne muscular dystrophy.