Access to eteplirsen may come in 2015; the company will conduct a confirmatory trial of eteplirsen and two additional trials of the drug
Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S. commercial demand for our lead product candidate eteplirsen in the event of an approval next year, the addition of internal resources willl enhance our ability to advance the development of our broader exon skipipng platform and explore the potential of our technology platform in other therapeutic areas."
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:
MDA's role in developing exon skipping
MDA has supported laboratory development of exon skipping since the 1990s and continues to support refinement of this strategy through grants to Steve Wilton at Murdoch (Australia) University and others. In addition, MDA provided supplemental funding for the phase 2 trial of eteplirsen conducted at Nationwide Children's Hospital in Columbus, Ohio. In addition, the Association's DMD Clinical Research Network's studies of disease progression in children under age 3 and in those who have stopped walking are likely to provide crucial information for the design of exon-skipping trials in these groups.
For more information
To learn more, see DMD: Exon-Skipping Timeline and Exon Skipping in DMD: What Is It and Whom Can It Help.