The European Commission has given conditional marketing authorisation for biopharmaceutical company PTC Therapeutics‘ Translarna (ataluren) in the EU.
Translarna is a treatment for the nonsense mutation-caused Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
With the authorisation, PTC can now sell Translarna in the 28 EU member states and the European economic area members Iceland, Liechtenstein and Norway.
PTC Therapeutics CEO Stuart Peltz 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."
PTC is now required to complete its confirmatory Phase III trial in nmDMD (ACT DMD) and submit additional efficacy and safety data from the trial.
The authorisation is based on the safety and efficacy results from a randomised double-blind multicentre study in 174 nmDMD patients for 48 weeks and additional retrospective analyses of study data from the company.
The study’s primary endpoint evaluated the effect of Translarna on ambulation as assessed by the change in distance walked (six-minute walk distance – 6MWD) during a six-minute walk test (6MWT).
Based on the post-hoc analysis, patients receiving Translarna (40 mg/kg/day given in three doses) had a 12.9m mean decline in 6MWD from baseline to week 48, and patients receiving placebo had a 44.1m mean decline in 6MWD.
In addition, patients receiving the treatment showed better improvement in secondary end points such as stair climb and stair descend time-function tests.