On June 27, 2013 GlaxoSmithKline announced that Drisapersen, a medication designed to treat Duchenne muscular dystrophy, had received breakthrough status form U.S. regulators. The designation of “breakthrough” status is certainly noteworthy, as the FDA created this new status at the beginning of 2013 to indicate medications that proved to offer significant benefits over other medications already offered. As an exon-skipping medication, Drisapersen has recently been touted for its performance in phase 2b clinical trials in boys afflicted with Duchenne muscular dystrophy, also coined DMD.
Thus far trials of this now breakthrough drug have indicated success for those boys who were treated with Drisapersen as compared to the boys who were administered a placebo. Of the 53 boys in the referenced 2b study, there were three different dosing groups: one group of boys who received a continuous dose of Drisapersen, one group of boys who were given intermittent doses of Drisapersen, and one group of boys who received a placebo. The group of boys who were given intermittent doses of Drisapersen were able to walk farther than the boys who were dosed with the placebo in trials. Even better results were attained with the group of boys who were dosed with continuous Drisapersen, as this clinical trial group walked substantially farther in six minutes as compared to the placebo group, when tested right after 24 weeks of treatment. At the 48 week mark in this trial the boys who were administered both intermittent and continuous doses of Drisapersen showed a 117 foot distance in walking ability as compared to the placebo group. Further results, particularly dystrophin protein levels are expected to be conveyed in October of 2013.
The effectual ability of Drisapersen to induce the exon 51 skipping in the DMD gene makes this now breakthrough therapy a potential future blockbuster drug. LGM Pharma provides Drisapersen CAS# 1251830-50-8, for research and development purposes. Other additional medications are under review as possible treatments for DMD as well, which LGM Pharma supplies. Clients can be assured of quality API products and continuous support throughout the R&D process.
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