Amyotrophic lateral sclerosis (ALS) remains a bewildering disease. Although it is rare, it is not uncommon. The lifetime risk of developing the condition is roughly 1 in 300 by age 85.
The heterogeneity of the disease is also unique, said Tammy Pighin Moore, CEO of the ALS Society of Canada. “About 5% to 10% of the people diagnosed with ALS have a familial or hereditary connection to it that we can easily understand because of the genes implicated,” Moore said.
Symptoms can begin in the limb (known as ‘limb onset’) or first affect speech or swallowing (known as ‘bulbar onset’). Some patients with the disease die months after diagnosis, while others may live for decades. For example, the famed physicist Stephen Hawking lived with the condition for 55 years. The average life expectancy after diagnosis is two to five years.
Health Canada recently became the first country to approve the ALS drug Albrioza (AMX0035) from Amylyx. The agency will require Amylyx to provide additional data from studies, including the ongoing Phase 3 PHOENIX trial.
In the following interview with Drug Discovery & Development, Moore discussed the potential of Albrioza and shared an overview of the treatment landscape and how ALS Society of Canada is working to secure reimbursement from payers for the drug.
DDD: Could you share your thoughts on the critical unmet need for new ALS treatments?
Moore: We anticipate that many therapies would be necessary because there are probably many forms of ALS.
We have riluzole, which was first approved in Canada in 2000.
Then, we had edaravone (Radicava), which Health Canada authorized in 2018, and finally became available to all Canadians in all provinces and territories in November 2021.
And now we have Albrioza, which has taken almost a year to go through the Health Canada process. We don’t have any line of sight of how long it will take to go through the decision process at the provinces and territories level or with other payers.
None of those therapies can cure the disease — they slow progression. They may slow progression for one person but not for another.
DDD: What role do you see Albrioza playing in treating ALS?
Moore: ALS clinical trials focus on a very defined subgroup of individuals with the disease. Sponsors have to ensure that they have statistical significance in the outcome measures. When we think about the number of people who potentially could benefit, we would like to see that it be available to more people because we don’t know who exactly may be a responder.
The other reality for people with ALS is that it can take up to several years even to get a definitive diagnosis. So you could move through a therapeutic window before ever even having your diagnosis.
Some studies from the Canadian Neuromuscular Disease Registry (CNDR) have shown that, even in Ontario, it can take 23.5 months to get an ALS diagnosis.
We want to make sure that people have access to these therapies when they become available and when they’ve got that diagnosis.
How does the ALS Society of Canada plan to work to get payer support for Albrioza?
Moore: We are working with companies even as they run their clinical trials to ensure they understand the Canadian landscape. Then, as soon as a company is considering a clinical trial and approaches Canada, we help coordinate the clinics so that we can have clinical trial access within the country. Because if a pharmaceutical company runs a clinical trial, they’re far more likely to consider that country for market access later on, should they have successful results.
That work starts years and years in advance, as it did with Amylyx. We had approached them many, many years ago.
As the company is looking to go through the Health Canada approval, we help them navigate that pathway and make appropriate contacts with the clinicians and whatever resources and groups are required.
We are also working to make sure the patient perspective is brought forward to decision-makers and a clinical perspective.
Last year, ALS Canada worked with the many parties within the community and created a position paper called “The time is now,” outlining that we feel that all ALS therapies should be available to people immediately upon Health Canada approval and let them negotiate beyond that. And companies can find creative ways to do it, and so can payers.
All Canadians should be able to get access immediately. If therapies can slow the progression of the disease, that can be so significant for somebody with ALS. Even if it means preserving a minor movement, such as being able to slightly move your hand, that means you could probably control your electric wheelchair, which could mean your entire independence. Preserving functionality and life expectancies for longer can be so meaningful to a person with ALS.
We specifically work within Ontario to ensure that there are other organizations and individuals prepared to lobby for each of their provincial and territorial payers to ensure that they are putting Albrioza in their provincial formularies as quickly as possible.