Boys with DMD continuously treated with eteplirsen for three years walked farther in six minutes than those receiving a placebo for the first six months
Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of...
Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
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.
Sarepta Therapeutics presented new favorable safety and efficacy results for its experimental exon-skipping drug eteplirsen, designed to treat Duchenne MD caused by specific dystrophin mutations
Update (Feb. 5, 2014): An analysis of pulmonary function tests at 120 weeks (2.3 years) found boys treated with eteplirsen showed a stabilization of respiratory function over a time period and in an age group where a decline would have been expected. Sarepta announced these results in a Feb. 5, 2014, press release.
Eteplirsen-treated boys with DMD who could perform the six-minute walk test showed continued stability in walking distance at week 120 of a phase 2b trial
Update (Jan. 16, 2014): Read Eteplirsen Data Still Strong: Treated Boys with DMD Stable at 120 Weeks for a recap of Sarepta's Jan. 15 presentation.
In a new webcast, Sarepta Therapeutics said it will seek approval for and start a confirmatory trial of eteplirsen in 2014; additional drugs may follow
Sarepta Therapeutics announced it is on track to submit an application to the U.S. Food and Drug Administration (FDA) for approval of eteplirsen in the first half of 2014.
In parallel with that application, the company is preparing to conduct a large-scale, confirmatory trial of eteplirsen. It plans to open the trial during the first quarter of 2014.