Biopharmaceutical company PTC Therapeutics (Nasdaq: PTCT) has received conditional marketing authorization from the European Commission for its drug Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.
Stuart Peltz, chief executive of PTC Therapeutics, said: “We are delighted that Translarna was approved for the treatment of nonsense mutation Duchenne muscular dystrophy. By targeting the underlying cause of DMD, it has the potential to change the course of the disease. We are moving rapidly to make this product available to patients in the EU as we continue our global efforts to fulfill our vision of making Translarna available to all the boys it may benefit.”
The approval will mean that PTC can market Translarna in the 28 member states of the European Union and European Economic Area, and is obliged to complete its confirmatory Phase III trial, submitting additional efficacy and safety data.
The primary endpoint of the trial, which had 174 patients, was to assess the change in distance walked during a six-minute walk test. The Translarna arm showed patients were able to walk 31.3 meters better than the placebo group. Patients on Translarna also showed a slower rate of decline in ambulation. The Committee for Medicinal Products for Human Use of the European Medicines Agency found that the results suggested Translana slows the loss of walking ability in these patients.
In terms of safety, serious adverse events were infrequent, and none were deemed to be caused by Translarna. The most frequent adverse reactions to the stated dose were nausea, vomiting and headache, and no patients discontinued with Translana due to an adverse reaction.