Health Canada starts review of PTC Therapeutics’ NDS for Translarna

The NDS will be reviewed with advance consideration under the Health Canada policy of Notice of Compliance with Conditions (NOC/c).

If approved, Translarna would be the first treatment available to patients in Canada that addresses an underlying cause of dystrophinopathy, including nonsense mutation Duchenne muscular dystrophy (nmDMD).

"We now have a better understanding about the different kinds of genetic mutations that can cause Duchenne muscular dystrophy and are making great progress in developing potential treatments that address the underlying genetic causes of the disease," said Dr. Craig Campbell , Head of Pediatric Neurology at London Health Sciences Centre.

"Every day matters for patients with nonsense mutation Duchenne muscular dystrophy. We look forward to the potential for a new treatment option that addresses an underlying cause of this relentlessly progressive disease which leads to the loss of walking ability in childhood, and premature death."

Health Canada’s NOC/c policy is available for promising new drug therapies intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases, or conditions for which no drug is presently marketed in Canada , or that represents a significant improvement in the benefit/risk profile over existing therapies. An NDS reviewed under the advanced consideration NOC/c policy is granted an expedited review in 200 days compared to standard NDS review of 300 days.

PTC Therapeutics Stuart Peltz CEO said: "There is a clear and urgent need for a treatment for people living with nonsense mutation Duchenne muscular dystrophy.

"We are grateful that Health Canada has recognized this need by granting Translarna an expedited review."

"This filing is a very important milestone for us and we look forward to working with Health Canada to bring Translarna to patients in Canada as soon as possible," said Michael Cloutier , General Manager of PTC Therapeutics in Canada.

"We are very pleased to have a new Canadian presence with our office in Burlington, Ontario , and are now fully operational in Canada."

The NDS for Translarna is based on the safety and efficacy results from a randomized double-blind multicenter study in 174 nmDMD patients for 48 weeks, and an additional retrospective analyses of study data. These data formed the basis for PTC’s marketing authorization application in Europe in 2014, which resulted in Translarna being licensed in the European Economic Area for the treatment of nmDMD in ambulatory patients aged five years and older. PTC Therapeutics is conducting a confirmatory Phase 3 randomized double-blind multicenter study in patients with nmDMD and the top line results from this study will be available in the fourth quarter of 2015.