On April 25, 2023, the U.S. Food and Drug Administration (FDA) approved Qalsody (tofersen) for the treatment of SOD1-ALS under the Accelerated Approval pathway. This blog post explores information on the therapy and discusses what this approval means to the Canadian ALS community. 

To help with certain terms, you can access our Research Glossary by clicking here. 


Last updated: May 1, 2023 

What is Qalsody (tofersen)? 

Qalsody (tofersen) is a prescription medicine used to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. The therapy is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. 

How is Qalsody (tofersen) administered? 

The treatment is administered intrathecally, a route of administration of drugs via injection into the spinal canal, via lumbar puncture, with a recommended dosage of 100 mg/15 mL per administration. Individuals receive three initial doses administered at 14-day intervals, followed by a maintenance dose every 28 days. Intrathecal injection is a routine procedure. 

What is the SOD1 gene? 

The SOD1 gene provides instructions for making a protein that helps protect our body’s cells from damage caused by harmful chemicals called reactive oxygen species. Mutations in this gene can cause a type of ALS that damages the nerve cells responsible for controlling movement, called motor neurons. In these cases, the abnormal protein made by the mutated gene is toxic to the motor neurons, leading to their death and resulting in the symptoms of ALS. 

Approximately two per cent of people living with ALS have a mutation in the SOD1 gene, the second most common cause of genetic ALS. Most individuals with a SOD1 mutation have a known family history of ALS. Some people without any obvious familial connection to ALS have tested positive for SOD1 mutations. 

Who can benefit from the treatment? 

Individuals must have a mutation in the SOD1 gene to benefit from the treatment.  

If you’re interested in learning more about the treatment, please get in touch with your ALS clinician, who will be able to interpret the clinical information about the drug as it pertains to individual situations. 

How do I know if I have an SOD1 mutation and could potentially benefit from Qalsody (tofersen)? 

In most situations, people with mutations in SOD1 will be aware of a family history of ALS, where multiple relatives have been diagnosed in the past. If you have a family history of ALS and are interested to know if it might be related to SOD1, please connect with an ALS clinician or research@als.ca for support in next steps.  

In some cases, SOD1 mutations have been found in people living with ALS who do not have an obvious family history. Much of the ALS field is evolving to genetic testing practices for all people with ALS, but this is not common practice everywhere. If you have ALS and are interested in finding out if you have a SOD1 mutation, please speak to your clinician or contact research@als.ca for support. 

An ongoing clinical trial with Qalsody (tofersen) is also searching for individuals with SOD1 mutations to be monitored for participation in a clinical trial where treatment can begin when the disease process has started but before any clinical symptoms have begun. A biomarker called neurofilament light has been established to detect the beginning of this underlying process and Qalsody (tofersen) will be tested to delay the onset of symptoms. If you have a known SOD1 related family history but do not have ALS, and are interested in learning more, please contact an ALS clinician or research@als.ca for support. 

If you have a known family history of ALS, do not know if it is linked to an SOD1 mutation, and are interested in learning more, please contact research@als.ca for support.  

ALS Canada aims to provide opportunities for more learning and information about SOD1-ALS in the days ahead, in conjunction with the Canadian ALS Research Network (CALS). It is important that we do everything possible to make sure anyone who stands to benefit from Qalsody (tofersen) is identified and able to access the treatment. 

Is there a trial for Qalsody (tofersen) in Canada? How can I participate? 

Canada has been involved in the clinical trials of Qalsody (tofersen) since the initial Phase 1 study. Participants from that study, as well as the completed Phase 3 (VALOR) clinical trial, are still accessing Qalsody (tofersen) through an ongoing open-label extension study. At this time, this open-label extension study is only available to individuals who participated in the clinical trial. 

There is an ongoing clinical trial of Qalsody (tofersen) for presymptomatic SOD1 carriers, recruiting at sites in Canada. This clinical trial is specifically for individuals who have specific SOD1 mutations but have not yet developed any symptoms of ALS. It requires regular monitoring to detect the initial stages of biological ALS occurring, before the onset of symptoms. More information about the study can be found at:

For further information, you can contact one of the Canadian trial sites listed here.  


What is the status of Qalsody (tofersen) within the drug access pathway in Canada? 

Qalsody (tofersen) is currently available to eligible individuals through Health Canada’s Special Access Program (SAP). Biogen, the pharmaceutical company that developed the treatment, has not yet begun the drug approval process in Canada.  

I know some people in Canada are taking this drug already. How can I access Qalsody (tofersen) in Canada? 

There are several Canadians who took part in the clinical trials and continue to access the therapy through open label extension (OLE) provided by Biogen. Additionally, Canadians have been able to access the therapy through the Special Access Program (SAP). 

Please speak with your ALS clinician for more information. 

When will Qalsody (tofersen) become available in Canada? 

Qalsody (tofersen) is currently available in Canada via Health Canada’s Special Access Program (SAP). Anyone diagnosed with SOD1-ALS should have access to the treatment through speaking to their clinician. You may also seek further information via research@als.ca. 

Biogen has not applied to Health Canada for approval of Qalsody (tofersen) at this time. 

What is the process for getting a drug approved through Health Canada? 

Health Canada conducts a review of the drug to assess its safety, efficacy, and quality. The process begins when the drug manufacturer files a New Drug Submission (NDS) with Health Canada. Health Canada must accept the New Drug Submission before the regulatory review begins.  

There are three timelines under which Health Canada conducts drug reviews: 

  • Standard Review, which is almost a one-year process (300 days) 
  • Notice of Compliance with Conditions, which is an approximately 200-day process 
  • Priority Review, which is a six-month process (180 days) 

Based on the review, Health Canada decides to either approve or reject the drug. If approved, the drug is granted a Notice of Compliance (NOC), which allows it to be sold in Canada and can be prescribed by physicians.  

We believe any new innovative ALS therapy should be granted priority review status. Due to the swift progression and terminal nature of the disease, people living with ALS cannot afford to wait almost a year for Health Canada to review a therapy. During this time, people will experience further loss of function, and ultimately, approximately 1,000 Canadians will die of ALS. Visit this blog post on How new drugs become approved and accessible to Canadians for more information. 

Can I buy the drug in the US and ship it to Canada? 

Qalsody (tofersen) is not yet approved by Health Canada. Individuals with SOD1-ALS who wish to access Qalsody (tofersen) should not need to obtain it in the United States and should ask their ALS clinician about the Health Canada Special Access Program. 


The approval of Qalsody (tofersen)  by the FDA in the United States is a positive development in our vision for a future without ALS and it highlights the importance of ongoing research investments, allowing the scientific community to pursue new discoveries that have the potential to transform ALS from a terminal disease to a treatable one. 

ALS Canada is in conversation with Health Canada and Biogen about the need for Canadians to have equitable access to this drug through the channels within the healthcare system, including the Special Access Program. We will continue to keep our community updated as we learn more.  

More information:

Travelling for treatment

Each person’s experience with ALS is unique. People living with ALS face different challenges and symptoms of the disease. It is important to be aware of these challenges and to consider both the benefits and risks when deciding on a treatment. This includes travelling abroad. Gathering as much information as possible will enable you to make well-informed decisions for you, and your loved ones.

When making the decision to travel abroad for medical treatments, some key considerations to be aware of both before you go, and when you return include things like, cost, safety, aftercare, rules for travelling with medications, immigration laws and visas.

Please speak with your ALS clinician for more information and consult your government’s healthcare authorities.

Posted in: Advocacy, Research