The investigational oral drug ataluren, in development to treat Duchenne muscular dystrophy (DMD) resulting from a specific type of genetic mutation, has received conditional approval in the European Union (EU). This designation allows patients to gain access to an experimental drug before it is fully approved. Ataluren, originally known as PTC124 and now bearing the brand name Translarna in Europe, is in development by PTC Therapeutics. The drug is not yet available in the United States except to participants in an ongoing, large-scale Phase III clinical trial. After the Phase III trial has been completed and if the results are favorable, PTC plans to submit an application for full approval of ataluren in the U.S. and the EU. The company estimates this submission will be in 2016.
