Sarepta's Duchenne drug gets a new date for FDA review
- The FDA has rescheduled its review panel for Sarepta Therapeutics muscle wasting drug eteplirsen to April 25, after it postponed the original review in January due to snowy weather in Washington, D.C.
- Sarepta's eteplirsen is in development for the treatment of a subset of patients with Duchenne's musculer dystrophy (DMD). Mainly affecting young men, DMD is a rare genetic disease which leads to progressive muscle wasting and usually proves fatal before the age of 30.
- In January, the FDA rejected another DMD treatment being developed by Biomarin, dashing the hopes of many patient advocates who were hoping for a new treatment. There are no drugs approved for DMD in the U.S. (Biomarin's drisapersen is still under review in the E.U.)
Sarepta has worked hard to generate compelling data for the FDA in line with standard clinical trial expectations, using historical control data to provide a context for analysis.
In October 2015, Sarepta announced positive results from a pivotal phase 2b trial in which treatment with eteplirsen resulted in a statistically significant advantage of 151 meters in the 6-minute walk test. In addition, the muscle biopsy data show an increased production of dystrophin, the missing protein needed to enhance muscle function in boys with DMD, in all of the patients.
However, FDA staff strongly questioned the persuasiveness of Sarepta's clinical trial data in a January report. The staff expressed its concerns about Sarepta's very small clinical trial size and the reliability of the 6-minute walk test.
The FDA will be reviewing Sarepta's data, in addition to other information culled from various sources, including patient advocates and parents of boys with DMD. The meeting, which is open to the public, will be held on April 25 from 8 am to 5:30 pm.