Sarepta and Eteplirsen

Posted by: Christine McSherry on October 20, 2012


The Duchenne Alliance and Jett Foundation are excited to share with you success of Sarepta Therapeutics RNA-based exon skipping compound, Eteplirsen. This innovative compound has the potential to treat a segment of those diagnosed with Duchenne, approximately 13% of the population. Data released yesterday by Sarepta’s CEO, Chris Garabedian, provided evidence that their lead candidate demonstrated a large increase inDystrophin-positive fibers, to 47% of normal on average, for the boys who were on the drug the entire 48 weeks of the trial. It is unclear if high dosing (50mg/kg) was more or less effective than the lower dosing (30mg/kg), however, it was apparent that there was an increase in Dystrophin-positivefibers the longer the participants were on the drug. The other endpoint that Sarepta announced yesterday was significant clinical benefit on the 6-minute walk test. The boys treated in the trial, all but two of them who became non-ambulatory during the trial, walked for longer distances. Lack of Dystrophin is the underlying cause of Duchenne muscular dystrophy.

What does this mean? It means that the course of this disorder can be modified by a compound. It means hope. Real tangible hope. Hope for the many individuals who can benefit from Eteplirsen, and hope for the many otherswho will benefit from a broader range of exon skipping compounds that are coming in the near future. Eteplirsen opens the doors for other exon skipping compounds. Our new challenge is to speed widespread access to this promising strategy. As we race to assist the 85% of those with amenable mutations, it’s important that we also give consideration to non-ambulatory individuals and women. This requires our community to unite to ensure there is solid preclinical data and to overcome manufacturing,regulatory, and clinical trial barriers.

Eteplirsen sets the new benchmark for Duchenne therapies and signals to researchers in academia and pharmaceutical and bio-tech companies that Duchenne is an alluring area where significant need exists and massive gains are possible. Now, more than ever, we need to work together to maximize our influence and impact. This is an urgent call to action to save our children. Now is the time to be united, “United to beat time.”

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