Prosensa Holding NV has announced that the U.S. Food and Drug Administration has outlined an accelerated regulatory approval path for its most advanced drug, aimed at treating a Duchenne muscular dystrophy (DMD). The Netherlands-based company plans to conduct two more studies, and file for U.S. marketing approval later this year, before filing for European approval in the near future. Like Sarepta Therapeutics Inc’s eteplirsen, Prosensa’s drug to treat DMD will probably win U.S. approval in 2015 at the earliest. Drisapersen, like eteplirsen, is designed to enhance the production of dystrophin, the lack of which causes DMD. DMD drugs-in-development have received encouraging signals from regulators in recent months. The FDA indicated an alternate path for approval to Sarepta’s eteplirsen in April, while European regulators recommended conditional approval for PTC Therapeutics Inc’s Translarna last month. Prosensa said in January it would pursue the development of drisapersen despite its failure in a late-stage trial, after additional data showed its use could slow disease progression. Accelerated approval is usually granted to drugs for serious diseases with no treatment options, based on data from initial trials. However, a company still needs to conduct larger trials to bolster its initial findings.
