PTC Therapeutics Inc. announced that the European Commission has granted conditional marketing authorization for Translarna (ataluren), in the European Union (EU) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. 

 

“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,” stated Stuart Peltz, CEO of PTC Therapeutics Inc. “We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. The DMD community has been waiting a long time for treatment options and this conditional approval marks an important day for us all.”

 

The authorization allows PTC to market Translarna in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway. As part of the conditional marketing authorization, PTC is obligated to complete its confirmatory Phase 3 trial in nmDMD (ACT DMD) and submit additional efficacy and safety data from the trial.

 

The approval is based on the safety and efficacy results from a randomized double-blind multicenter study in 174 nmDMD patients for 48 weeks and our additional retrospective analyses of study data. The 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). The post-hoc analysis showed that from baseline to Week 48, patients receiving Translarna (40 mg/kg/day given in 3 doses) had a 12.9 meter mean decline in 6MWD, and patients receiving placebo had a 44.1 meter mean decline in 6MWD. Thus the mean change in observed 6MWD from baseline to Week 48 was 31.3 meters better in the Translarna group than in the placebo group (p=0.056). Furthermore, in more severely affected patients whose baseline 6MWD was less than 350 meters, the mean change in observed 6MWD from baseline to Week 48 was 68 meters better in the Translarna group than in the placebo group. Patients on Translarna also showed a slower rate of decline in ambulation based on an analysis of time to 10% worsening in 6MWD. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency found that these results suggest that Translarna slows the loss of walking ability in nmDMD patients. 

 

Additionally, based on a retrospective analysis, patients receiving treatment also trended better in secondary endpoints such as stair climb and stair descend time-function tests, which the CHMP also found to suggest slowing of nmDMD progression relative to placebo. Safety results showed that Translarna was generally well tolerated. Serious adverse events were infrequent, and none was considered to be related to Translarna. The most frequent adverse reactions at the recommended dose were nausea, vomiting, and headache. These adverse reactions generally did not require medical intervention, and no patients discontinued Translarna treatment due to any adverse reaction. 

 

“The world’s first approved treatment for the underlying cause of DMD marks a very important moment for patients and their families. It is our highest priority to make Translarna available to patients and we will be working with regulators, payers, physicians and patient organizations to make that a reality.” stated Mark Rothera, chief commercial officer, PTC Therapeutics Inc. 

 

Date: August 4, 2014

Source: PTC Therapeutics