Health Canada Accepts New Drug Submission for tofersen

Today, Biogen Canada announced that Health Canada has accepted its New Drug Submission (NDS) for tofersen for the treatment of ALS in adults who have a pathogenic variant (also known as a mutation) in the superoxide dismutase 1 (SOD1) gene. This step triggers a thorough regulatory review of the therapy, which will result in the decision as to whether tofersen will be approved for use in Canada. 

Although a positive step forward, we are disappointed that tofersen was not granted a six-month Priority Review. As the ALS community knows well, the process for how drugs become accessible to Canadians takes far too long – and our position is that all ALS therapies should be given Priority Review by Health Canada, which would cut the time for this process in half.  

If approved, tofersen will be the first therapy indicated in Canada to target a genetic cause of ALS. Therefore, pathways that advance the pace at which proven therapies become approved and available to Canadians are necessary to address the urgent unmet need of people living with ALS. As this therapy is currently available through a Special Access Program (SAP), it is important that there are no gaps in access during this process. 

We will be connecting with Health Canada to seek clarification and further insights.  

ALS Canada will continue to provide updates as information becomes available. To learn more about tofersen, visit our frequently asked questions blog post on this topic. 

You can read more in Biogen’s press release and learn more about how drugs become approved and accessible in Canada by visiting our blog post on this topic.