Eteplirsen Still Proves to be Efficacious as Duchenne Drug


duchenne-msSarepta Therapeutics shared optimistic information in July 2014 with the news that Phase II Eteplirsen trial participants were continuing to prosper. The Phase II study of Eteplirsen is closing in on its third year for the boys with Duchenne muscular dystrophy, but the positive results are far from over. With such outstanding long term study results Sarepta is seeking to obtain an accelerated approval from the FDA. While a slight decline was reported in the 6 minute walk tests of 33.2 meters, results from this 144 week extension study are still promising. Data gleaned has revealed significantly improved performance for the boys in comparison to the placebo arm of the study participants who were transitioned to Eteplirsen after 6 months’ time. Sarepta executives fought back against criticism of the small decline in walk results by restating that Eteplirsen is not a cure for Duchenne muscular dystrophy {DMD}, but it is an effective treatment to aid the body in generating dystrophin. Thus far Eteplirsen has proven to be safe and tolerable for the participants with no major adverse effects  reported.

Research and development of medications to treat the symptoms of Duchenne muscular dystrophy are crucial for the 300,000 boys worldwide who are suffering from this debilitating condition. As a severely destructive muscle disease DMD shortens the life span of these young men and plagues their quality of life. Previous studies of Eteplirsen have proven the drug’s ability to induce dystrophin production and greatly improve walk test distances. Typical dosages of Eteplirsen were 30 or 50 mg/kg/wk. given as intravenous infusions.

LGM Pharma is a provider of Eteplirsen, CAS #1173755-55-9 for research and development purposes. Clients can be assured of quality API products and continuous support throughout the R&D process.

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