ALS Canada Advocacy Update – January-March 2024

This blog series is a place for the ALS community to learn about and stay updated on ALS Canada’s advocacy efforts as well as relevant developments within government. Please note that because ALS Canada advocates federally and provincially within Ontario, these updates will primarily feature updates from the federal and Ontario provincial levels of government.

Therapeutics updates

Masitinib

• 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.

ALBRIOZA (AM00X35)

• Amylyx Pharmaceuticals announced that the PHOENIX Phase 3 clinical trial of AMX0035, marketed as ALBRIOZA in Canada, did not meet its primary or secondary endpoints.
  • ALBRIOZA is approved by Health Canada under the Notice of Compliance with Conditions (NOC/c) pathway, with conditions depending on the outcome of the PHOENIX trial.

Tofersen

• Biogen Canada announced that Health Canada 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.
• 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.

Access to therapies

Meeting with the new pCPA leadership

• As a member of the Health Charities Coalition of Canada (HCCC) and HealthPartners, ALS Canada participated in a meeting with the first-ever CEO of the pan-Canadian Pharmaceutical Alliance (pCPA), Douglas Clark, to discuss pCPA’s top priorities and share our community’s perspective to inform the organization’s strategic plan.

Canadian Organization for Rare Disorders (CORD) – Rare Disease Day 2024 Summit

• To mark Rare Disease Day on February 29, ALS Canada attended the Canadian Organization for Rare Disorders’ Rare Disease Day 2024 Summit in Ottawa. During this time, we provided input on the experience of the ALS community during the multistakeholder expert panel discussing creating expanded, accelerated, and sustainable access to rare disease therapies.

Provincial Updates

ALS Canada’s Queen’s Park Day of Action

• On February 21, members of the ALS community joined ALS Canada at Queen’s Park, urging the Ontario government to invest $6.6 million of crucial funding in Budget 2024 to implement the Ontario Provincial ALS Program to support Ontarians living with ALS and their families with their clinical care and support needs.
• With participation from more than 40 MPPs, including the Minister of Health, Sylvia Jones, ALS Canada’s Queen’s Park Day of Action raised awareness of the current realities of the province’s health care system, which fails to meet the complex and urgent needs of people living with ALS, leaving more than 1,300 Ontarians without adequate care and support.

2024 Ontario Budget

• On March 26, 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.
  • Learn more about our 2024 Ontario Budget submission.

Government Meetings

• Throughout January and February, ALS Canada met with several Ontario MPPs, calling on the provincial government to implement the Ontario Provincial ALS Program in Budget 2024 to ensure Ontarians living with ALS and their loved ones receive the care and support they rightly deserve:
  • Doug Ford, Premier of Ontario
  • Office of the Minister of Health, Sylvia Jones
  • MPP Dawn Gallagher Murphy, Parliamentary Assistant to the Minister of Health
  • MPP David Smith, Parliamentary Assistant to the Minister of Labour, Immigration, Training and Skills Development
  • Raymond Cho, Minister for Seniors and Accessibility

Note to readers: This blog is a source of information for the ALS community that provides an overview of advocacy efforts and current affairs for Canadians affected by ALS. While there are many topics presented in this blog update, they don’t represent ALS Canada’s official position on any particular issue, nor indicate a complete list of ALS Canada’s advocacy priorities. We’re also unable to share all details about certain engagements with industry stakeholders due to confidentiality reasons.