DMD: Sarepta Will Pursue Accelerated Approval for Eteplirsen and Conduct Additional Trials


Sarepta Therapeutics, developer of experimental Duchenne muscular dystrophy (DMD) drug eteplirsen, says it will move forward as quickly as possible with a new drug application for this compound, as well as with a large-scale, confirmatory trial, two additional trial of eteplirsen, and trials of at least one other experimental drug of this class. 
Eteplirsen is an exon-skipping compound that targets exon (section) 51 of the dystrophin gene, coaxing muscle cells to leave out (skip) that section of genetic instructions and produce functional, though short, dystrophin protein molecules. It's been estimated that 13 percent of boys with DMD — those with dystrophin mutations near exon 51 — can potentially benefit from skipping exon 51. 
Sarepta summarized its current plans for eteplirsen and other exon-skipping drugs for DMD in a May 13, 2014, webcast for the DMD community, now archived on the company's website; and in a May 8, 2014, press release. 
Summary of the May 13 webcast
On the May 13 webcast, Sarepta made the following statements: 
The U.S. Food and Drug Administration (FDA), reversing an earlier decision, will consider an accelerated approval pathway for eteplirsen.
If the FDA grants an accelerated approval status for eteplirsen based on the phase 2b,12-participant trial results, the drug could become available to patients in 2015, although Sarepta would be required to conduct a confirmatory study to obtain full approval for the drug.
Sarepta is planning a confirmatory trial of eteplirsen for the third quarter of 2014, using a "historical" control group instead of a placebo group for comparison to an eteplirsen-treated group. Historical control groups comprise previously studied patients. 
The confirmatory trial of eteplirsen will include boys with DMD who are between 7 and 16 years old, can walk a minimum distance, and have dystrophin mutations potentially treatable by skipping exon 51.
In addition to the large-scale, confirmatory trial of eteplirsen in ambulatory (walking) boys, the company will test the drug in two other populations beginning in the fourth quarter of 2014: boys with DMD who are less than 7 years old, and those who can no longer walk a minimum distance and are 20 years old or younger.
By early 2015, Sarepta plans to open placebo-controlled studies of its exon-skipping compound targeting exon 45 and/or its compound targeting exon 53 in North America and Europe.
The company plans to begin an open-label (no placebo group) study of its compound to target exon 53 in Europe and the United Kingdom in the third quarter of 2014.
Details of these and other upcoming trials, including study sites and criteria, are not yet available but will be posted on and Sarepta's Let's Skip Ahead site when they are. Sarepta urges families to provide information and sign up for updates on the Let's Skip Ahead site under "Join Us."


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