Health Canada Initiates Review Of PTC Therapeutics' NDS For Translarna

(RTTNews) - PTC Therapeutics International, Ltd., a subsidiary of PTC Therapeutics, Inc. (PTCT), announced that Health Canada has initiated their review of the company's New Drug Submission or NDS for Translarna or ataluren to treat nonsense mutation dystrophinopathy.

The NDS will be reviewed with advance consideration under the Health Canada policy of Notice of Compliance with Conditions or 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 or nmDMD.

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.

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.