PTC withdraws the regulatory filing in Canada so they can supplement the phase II data with the phase III, ACT-DMD data.
PTC is having ongoing discussions with Health Canada (the equivalent of the FDA in Canada) during the review of their NDS (the equivalent of the FDA’s New Drug Application; NDA). PTC plans to submit the results of the phase III, ACT DMD study to the Canadian agency for review. In order to submit this additional data for review, PTC will withdraw the current NDS from Health Canada and resubmit the NDS with the ACT DMD results. The company expects to have a new pre-NDS filing meeting with Health Canada in the first half of 2016
Has PTC received a Notice of Deficiency from Health Canada?
No. In fact, PTC has withdrawn the NDS for Translarna in order to resubmit the NDS with the data from the ACT DMD study and plans to refile in the near future.
Why did PTC submit the NDS in Canada in September ahead of the results of the ACT DMD study? Why didn’t the company wait to file and include the ACT DMD study results?
PTC’s priority has always been to get Translarna to Duchenne patients as quickly as possible and they submitted their NDS in Canada based on the results of our P2b clinical study. They had a pre-NDS meeting with Health Canada and based on interactions with Health Canada at that meeting and the fact that the EMA approved Translarna based on the P2b data, they determined that filing an NDS for review with advance consideration under the Health Canada policy of Notice of Compliance with Conditions (NOC/c) provided the quickest approach to get Translarna to patients in Canada.