Amylyx Pharmaceuticals announced that they have started a process with Health Canada to discontinue the marketing authorization for ALBRIOZA. This means that ALBRIOZA will no longer be available for new patients in Canada. People currently on the therapy in Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug program.
ALBRIOZA was approved by Health Canada under the Notice of Compliance with Conditions (NOC/c) pathway in 2022. Amylyx’s decision to remove ALBRIOZA from the market is based on the recent PHOENIX trial results of AMX0035, marketed as ALBRIOZA in Canada and RELYVRIO in the United States, which failed to show an effect on primary and secondary outcome measures.
While we know that this news is disappointing for the ALS community and brings uncertainties, we also hold onto hope for the future. Research into ALS treatments is ongoing, with researchers tirelessly working to uncover new insights and potential treatments. Each advancement brings us one step closer to understanding this complex disease and, ultimately, paving the way for effective treatments that can improve the lives of people affected by ALS.
The ALS Society of Canada remains committed to playing a significant role in the collective effort to drive progress and innovation in ALS research with the vision of a world free of ALS and continue advocating for equitable, timely, and affordable access to approved therapies.
We encourage people living with ALS and their families to consult with their clinicians for guidance during this transition.
Today, Ontario’s Minister of Finance, Peter Bethlenfalvy, presented the province’s 2024 Budget. While the initial budget announcement did not explicitly include a $6.6 million investment to implement the recommendations outlined in the Ontario Provincial ALS Program, we will continue our discussions with the government and relevant ministries to seek clarification. We look forward to understanding how our ask is considered in the 2024 Budget through the funding letters released in the coming weeks.
We will continue to keep you informed of any developments and appreciate your continued support.
Learn more about our 2024 Ontario Budget submission.
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.
Amylyx Pharmaceuticals has announced that the PHOENIX Phase 3 clinical trial of AMX0035 did not meet its primary or secondary endpoints.
The results from the 48-week study, which involved participants from the United States of America and Europe, showed no significant benefit from AMX0035 compared to placebo, as measured by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. AMX0035 was well-tolerated, and the safety profile was found to be consistent with previous studies.
AMX0035, marketed as ALBRIOZA in Canada, was approved by Health Canada under the Notice of Compliance with Conditions (NOC/c) pathway, with conditions depending on the outcome of the PHOENIX trial.
The ALS Society of Canada will work with clinicians, regulators and other key experts to better understand the implications of these results for Canadians living with ALS.
We know that this news is disappointing for the ALS community, and we will continue to provide updates as more information becomes available.
We appreciate Amylyx’s commitment to the ALS community, and their responsible and transparent approach to providing clear information about the study results.
The ALS Society of Canada extends its deepest gratitude to all people and families affected by ALS who participated in this trial. Their involvement in the trial has significantly contributed to our understanding of the disease and informs future research.
Statement from Canadian ALS Research Network on results from the PHOENIX Trial
Members of the Canadian ALS Research Network (CALS), representing the leadership of Canada’s multidisciplinary ALS clinics, were disappointed to learn of the negative results from the PHOENIX Phase 3 clinical trial of AMX0035.
The results from the 48-week study, involving participants from the United States of America and Europe, revealed no significant benefit from AMX0035 compared to placebo, as measured by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. AMX0035 was well-tolerated, and the safety profile was consistent with previous studies; however, there was no evidence for slowing of disease progression.
AMX0035, marketed as ALBRIOZA in Canada, was approved by Health Canada under the Notice of Compliance with Conditions (NOC/c) pathway, with conditions depending on the outcome of the PHOENIX trial.
CALS Members understand that these results may be a confusing outcome given the positive findings in a previous Phase 2 trial. The Phase 3 trial, being larger and more rigorous, plays a critical role in confirming or refuting earlier findings, and concluded there was no significant benefit from AMX0035 (ALBRIOZA) compared to placebo.
In light of these findings, CALS physicians recommend discontinuing the use of AMX0035 (ALBRIOZA) for the treatment of ALS at this time. We appreciate the support that Amylyx has given the ALS community, and their responsible approach to providing clear information about the study results.
The negative results of the trial are a frustrating outcome for patients, families, and ALS clinicians. However, we remain hopeful that new treatments for ALS will emerge in the near future.
AB Science announced that Health Canada has issued a Notice of Non-Compliance–Withdrawal (NON-W) for masitinib in ALS due to unresolved clinical concerns and data reliability issues.
A Notice of Non-Compliance-Withdrawal (NON-W) indicates that the drug submission did not meet the necessary standards and requirements set by Health Canada, resulting in the review process being incomplete and non-compliant at the time of issuance.
AB Science has the opportunity to submit a request for reconsideration within 30 days, during which Health Canada would re-examine the decision based on additional data provided with the goal to ensure that masitinib meets the necessary regulatory standards for ALS treatments in Canada.
We will continue to post updates as we become aware of them. You can read more in AB Science’s press release. Specific questions about the treatment can be directed to the ALS Canada Research Team at research@als.ca.
The province of Manitoba has listed RADICAVA Oral Suspension on the Manitoba Pharmacare Program (Exceptional Drug Status) as a treatment for ALS. With this announcement, all provinces and territories across Canada – as well as Veterans Affairs Canada and Indigenous Services Canada – are now covering RADICAVA Oral Suspension through their public formularies. More than 700 Canadians helped make this possible by writing their provincial Health Ministers urging the province to make a quick decision in favour of covering the drug, and we are pleased that affordable access to RADICAVA Oral Suspension is now available to all eligible Canadians regardless of where they live. For more information, you can read Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA)’s press release.
While compared to the ALS community’s experience with Radicava (edaravone)’s approval and reimbursement process in 2021, we are seeing improvements to the timelines associated with access. However, time is of the essence for the ALS community. The timeframe between approval of the therapy and for all provinces to make a reimbursement decision does not reflect the realities of living with ALS. RADICAVA Oral Suspension was approved by Health Canada in November 2022. It took over one year from Health Canada approval for all provinces, territories, and federal programs to cover the therapy. Every day counts for Canadian families living with the disease, which is why we want to see a streamlined process where ALS therapies get to Canadians in a timeframe that more accurately reflects the urgency faced by the community.
ALS Canada will continue actively working with federal and provincial decision-makers to ensure Canadians living with ALS have equitable, timely, and affordable access to Health Canada approved therapies.
The province of Ontario has listed RADICAVA Oral Suspension (edaravone), also known as oral edaravone, on the public formulary as a therapy accessible through the Ontario Drug Benefit Program’s Exceptional Access Program (EAP) as a treatment for ALS. This decision comes quickly after the conclusion of the pCPA negotiations, making Ontario the first province to fund oral edaravone, demonstrating that together the collective voices of the ALS community can make a difference.
We urge all remaining provinces and territories to reimburse oral edaravone without delay.
Now more than ever, advocating to your provincial and territorial government will send a clear message to decision-makers that there is an urgent need for the ALS community to have equitable access to treatments. Advocating for swift and equitable public reimbursement of oral edaravone will help lay the groundwork for access to future ALS therapies. It can take as little as 60 seconds to tell the Minister responsible for health in your province or territory that oral edaravone must be reimbursed without delay.
ALS Canada will continue to actively support the other provincial drug programs to make informed, inclusive, and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS.
If you live in Ontario and are interested in accessing the therapy, connect with your ALS neurologist to understand any conditions for coverage.
Last updated: November 7, 2023
July 20, 2023 – Mitsubishi Tanabe Pharma Canada and the pan-Canadian Pharmaceutical Alliance (pCPA) have successfully concluded negotiations for RADICAVA Oral Suspension (edaravone), also known as oral edaravone, and entered into a Letter of Intent (LOI) for the terms and conditions under which the therapy would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada.
It is now up to individual provinces and territories to make a reimbursement decision for the therapy. If negotiations are successful, provincial and territorial drug plans will provide reimbursement for oral edaravone.
While this is a positive step forward in access to therapies, time is of the essence for the ALS community. Canada’s drug access and reimbursement processes and timeframes do not reflect the urgency of people living with a terminal disease. Oral edaravone received approval from Health Canada in November 2022. In the 10 months it took for the pCPA negotiations to conclude, it’s estimated that hundreds of Canadians living with ALS will have died while waiting for potential access to the therapy and hundreds more will die if they must continue to wait months or years for the decision by each province and territory to cover the therapy under provincial drug programs.
ALS Canada will continue to actively support the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS. We are hopeful that provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for all Canadians who may benefit. There is simply no time to wait for people affected by ALS.
Advocating for swift public reimbursement of oral edaravone will help lay the groundwork for access to future ALS therapies. It can take as little as 60 seconds to tell the Minister responsible for health in your province or territory that oral edaravone must be reimbursed without delay. Participate in the campaign here.
As of November 7, 2023, following provinces have made decisions regarding the public reimbursement of RADICAVA Oral Suspension (edaravone):
- Ontario: Under the Ontario Drug Benefit Formulary (Exceptional Access Program)
- Alberta: Under the Alberta Drug Benefit List
- Quebec: Under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
- British Columbia: Under the British Columbia PharmaCare (special authority)
- New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary (special authorization)
- Nova Scotia: Under the Nova Scotia Formulary (exceptional status)
- Saskatchewan: Under the Saskatchewan Drug Plan Formulary (Exception Drug Status)
- Newfoundland and Labrador: Under the Newfoundland and Labrador Prescription Drug Program (NLPDP) (special authorization)
- Prince Edward Island: Under the Prince Edward Island (PEI) Pharmacare Formulary (special authorization)
We will continue to post updates as they become available. You can read more in Mitsubishi Tanabe Pharma Canada’s press release and learn more about how drugs become approved and accessible in Canada by visiting our blog post on this topic.
Updated on November 27, 2023
June 15, 2023 – Amylyx and the pan-Canadian Pharmaceutical Alliance (pCPA) have successfully concluded negotiations for ALBRIOZA and entered into a Letter of Intent (LOI) for the terms and conditions under which ALBRIOZA would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada.
It is now up to individual provinces and territories to make a reimbursement decision for the therapy. If negotiations are successful, provincial and territorial drug plans will provide reimbursement for ALBRIOZA.
While this is a positive step forward in access to therapies, time is of the essence for the ALS community. Canada’s drug access and reimbursement processes and timeframes do not reflect the urgency of people living with a terminal disease. ALBRIOZA received approval with conditions from Health Canada in June 2022. In the year it took for the pCPA negotiations to conclude, an estimated 1,000 people living with ALS have died while waiting for access and thousands more will die if they must continue to wait months or years for the decision by each province to cover the therapy under provincial drug programs.
ALS Canada will continue to actively support the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS. We are hopeful that provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for all Canadians who may benefit. There is simply no time to wait for people affected by ALS.
Advocating for swift public reimbursement of ALBRIOZA will help lay the groundwork for access to future ALS therapies. It can take as little as 60 seconds to tell the Minister responsible for health in your province or territory that ALBRIOZA must be reimbursed without delay.
As of November 27, 2023, following provinces have made decisions regarding the public reimbursement of ALBRIOZA:
- Ontario: With criteria under the Ontario Drug Benefit Formulary (Exceptional Access Program)
- Québec: With criteria under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
- British Columbia: Under BC PharmaCare Formulary (special authorization)
- New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary
- Alberta: Under the Alberta Drug Benefit List
- Nova Scotia: Under the Nova Scotia Pharmacare Program
- Newfoundland and Labrador: Under the Newfoundland and Labrador Prescription Drug Program (NLPDP)
- Manitoba: Under the Manitoba Drug Benefits and Interchangeability Formulary (special authorization)
- Saskatchewan: Under the Saskatchewan Drug Plan Formulary
- Prince Edward Island: Under the PEI Pharmacare Formulary
Today, the Federal Government announced the National Strategy for Drugs for Rare Diseases. We are pleased to see this strategy move forward and hope it will be a significant step in building knowledge and improving access to therapies for people living with rare diseases, including the ALS community.
Throughout the establishment of the strategy, the ALS Society of Canada had the opportunity to bring forward the perspectives of the ALS community. We remain committed to being a partner and working with the federal and provincial governments to establish rare disease pathways in each province, while advocating for swift action to meet the urgent needs of people affected by ALS. We will also continue to engage with all stakeholders on various initiatives to support the implementation of the strategy.
“Every day counts for Canadian families living with ALS and we cannot continue to wait years through a series of drawn-out, confusing, red-tape-filled processes for access to innovative therapies. With the announcement of the National Strategy for Drugs for Rare Diseases, we call on each province to utilize the Government of Canada’s investments and establish a pathway for expedited and equitable access to therapies today and in the future. The Time is Now to support all Canadians living with ALS.” – Tammy Moore, CEO, ALS Canada
We will continue to share information as we learn more about the National Strategy for Drugs for Rare Diseases.
You can read the government’s press release here.
You can read our roadmap to a streamlined pathway for expedited and equitable access to ALS therapies in our position paper The Time is Now.
Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA), a subsidiary of Mitsubishi Tanabe Pharma America, Inc. (MTPA), announced that Health Canada has approved the oral formulation of edaravone. This decision came after a 180-day priority review of the drug’s safety, efficacy and quality.
Health Canada’s approval of oral edaravone under a priority review pathway underscores the importance of equitable and expedited access to effective treatments. However, at this stage in the drug access processes, the drug is not yet covered through private or public drug programs. CADTH (and INESSS in Quebec) must complete its review of the therapy and then private, and provincial public drug plans must decide if they will cover the costs associated with the drug.
ALS Canada will continue to bring forward the perspectives of people affected by ALS and encourage CADTH, the pan-Canadian Pharmaceutical Alliance (pCPA) and the provincial drug programs to make informed reimbursement decisions in a timeframe that more accurately reflects the reality of this devastating disease.
You can read more in MTP-CA’s press release and learn more about how drugs become approved and accessible in Canada by visiting our blog post on this topic.
Today, Amylyx Pharmaceuticals, Inc. announced that Health Canada has approved ALBRIOZA (AMX0035) under a Notice of Compliance with Conditions (NOC/c). This means the drug will soon be made available and can be marketed in Canada with certain conditions.
Health Canada’s approval is a positive step forward in the available treatment options for people living with ALS. However, at this stage in the drug access processes, the drug is not yet covered through private or public drug programs. CADTH (and INESSS in Quebec) must complete its review of the therapy and then private, and provincial public drug plans must decide if they will cover the costs associated with the drug.
ALS Canada will continue to be involved in the CADTH Health Technology Assessment process and is actively working to support the pan-Canadian Pharmaceutical Alliance (pCPA) and the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS. We are hopeful that pan-Canadian and provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for all Canadians who may benefit. There is simply no time to wait for people affected by ALS.
TORONTO, May 13, 2022 – Today, Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA), a subsidiary of Mitsubishi Tanabe Pharma America, Inc. (MTPA), announced that Health Canada has accepted its filing of a Supplement to a New Drug Submission (SNDS) for an investigational oral formulation of edaravone. This step triggers a thorough regulatory review of the therapy, which will hopefully result in the drug being approved for use in Canada.
We are also very encouraged to learn that Health Canada has granted a Priority Review, which expedites the review timeline for the therapy to six months.
We believe any new innovative ALS therapy should be granted priority review status – and we are hopeful this decision sets an important precedent for all future ALS therapies. 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.
You can read more in MTP-CA’s press release and learn more about how drugs become approved and accessible in Canada by visiting our blog post.
The province of Prince Edward Island (PEI) has listed Radicava (edaravone) on the public formulary under Special Authorization as a treatment for ALS. With this announcement, all provinces and territories across Canada – as well as Veterans Affairs Canada and Indigenous Services Canada – are now covering Radicava through their public formularies. More than 3,600 Canadians helped make this possible by writing their provincial Health Ministers urging the province to make a quick decision in favour of covering the drug, and we are pleased that affordable access to Radicava is now available to all eligible Canadians regardless of where they live. You can read Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA)’s press release for more information.
While a positive step forward in access to ALS therapies, time is of the essence for the ALS community. The timeframe for all provinces to make a reimbursement decision was far too long. Radicava was approved by Health Canada in October 2018. It took over three years from Health Canada approval for all provinces to cover the therapy through their public drug plan. Every day counts for Canadian families living with the disease, which is why we want to see a streamlined process where ALS therapies get to Canadians in a timeframe that more accurately reflects the urgency faced by this community.
Our recently published position paper, The Time is Now, reflects on the experience with Radicava and explores the complexity of Canada’s drug access processes, proposing two solutions to get Health Canada approved therapies to Canadians living with ALS faster.
Make your voice heard! The Canadian Agency for Drugs and Technology in Health (CADTH) has posted a call for patient input on AMX0035. ALS Canada will be preparing a submission reflecting the perspectives of the ALS community and we are inviting you to contribute. In early November, you will have the opportunity to participate in a survey where you can share your perspectives, experiences and expectations for the therapy.
Your input will help to ensure that issues of importance to patients are considered by decision-makers at CADTH, who ultimately recommend whether public drug plans should cover the drug if it is approved by Health Canada. A concurrent Health Canada and CADTH review is just one step of many in aligning the drug access processes to the urgent needs of the ALS community.
At this time, Health Canada has not yet finished its review of AMX0035, so a decision about its approval is still to come. You can learn more about how drugs become accessible in Canada by visiting our blog post on this topic.
November 1, 2021 – The Phase 3 VALOR results for tofersen did not show a statistically significant change in disease progression as assessed by the ALSFRS-R scale, however a number of other outcome measures suggested the treatment was able to have an effect on people with SOD1-ALS. As more open label extension data is shared, and further discussion across the field occurs, there will be a stronger consensus on what the data reveals about tofersen.
In other promising news related to this therapy, Biogen recently initiated the ATLAS study, which will start treatment of tofersen in people living with an SOD1 mutation before symptoms of ALS begin. The study will look at whether early treatment with tofersen can significantly delay the onset of clinical symptoms. Participants randomized to placebo would be provided active drug immediately upon diagnosis. Never clinically tested before, early intervention has long been considered a key area for study in ALS. The ATLAS trial is an important next step not only in the evolution of tofersen for SOD1-ALS but in paving the way for future ALS trials.
Read more in the International Alliance of ALS/MND Associations Scientific Advisory Council (SAC) briefing note on tofersen.
October 19, 2021 – The ALS community has been eagerly anticipating the results of the Phase 3 VALOR trial for tofersen, an experimental drug being evaluated as a therapy for a genetic form of ALS caused by mutations in the gene SOD1. The results were presented earlier this week at the American Neurological Association (ANA) 2021 virtual meeting.
Although the primary endpoint of ALSFRS-R was not met in the same manner many anticipated, the data does provide some hopeful signals. Other data from the study showed positive indications that the therapy may affect disease progression but further discussion and analysis across the field will help to better understand the complexity of the results and what this means for next steps. The many people and families around the world affected by SOD1-ALS who participated in the study deserve incredible thanks for their contributions. More information to come. You can read the Biogen press release here.
