Amylyx agrees to deal for Albrioza reimbursement in Canada for ALS
Approved treatment for ALS also available in US as Relyvrio
Albrioza, the approved treatment for amyotrophic lateral sclerosis (ALS) formerly known as AMX0035, is one giant step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Amylyx Pharmaceuticals.
Amylyx has reached an agreement with the pan-Canadian Pharmaceutical Alliance, or pcPA — a government partnership in Canada that collaborates on various public drug plan initiatives — for terms under which ALS patients in the country can be reimbursed for Albrioza.
The negotiations began after the therapy, marketed as Relyvrio in the U.S., was conditionally approved by Canadian authorities a year ago. While Albrioza became commercially available in the country in August 2022, it hasn’t yet been listed on most public health plans — meaning patients on those plans couldn’t be reimbursed for its costs.
Amylyx has now signed off on its intentions to honor the new agreement in a letter of intent (LOI), which, if finalized, will enable eligible patients to avoid high out-of-pocket costs for the medication.
“Today marks a major milestone for people living with ALS and their caregivers in Canada. With this signed LOI, eligible Canadians living with this rapidly progressive disease who rely on public coverage are one step closer to accessing Albrioza,” Chris Aiello, general manager and head of Canada at Amylyx, said in a company press release, adding, however, “Our work is by no means done.”
Advocates cite lengthy delay in getting treatment for ALS covered
With the LOI now in place, the company will move forward to work individually with federal, provincial, and territorial drug plans to finalize the agreements, which will enable Albrioza to be listed for reimbursement on those plans.
Although the news is welcome, some believe it did not come fast enough for patients, many of whom see rapid declines in the first few years after disease onset.
“While it is good news that Canadians living with ALS are closer to accessing this innovative treatment, some people living with ALS have been waiting a year to access this therapy, and they will continue to wait,” said Tammy Moore, CEO of the ALS Society of Canada.
“People with ALS do not have the luxury of time,” Moore said.
We believe there are opportunities to streamline the multiple steps and lengthy processes involved in achieving public reimbursement for treatments like Albrioza in Canada. The time is now for equitable and expedited access to approved treatments for ALS, and we will continue advocating on behalf of the ALS community to improve their access to care.
The process of a therapy being listed on public health plans involves sometimes lengthy discussions between pcPA and the company that markets the therapy. The pcPA has to negotiate the price of the treatment on behalf of public drug payers, including both federal and local governments, along with determining which patients will be eligible for reimbursement.
Additional discussions then ensue with each individual public drug payer to work out the details.
“We believe there are opportunities to streamline the multiple steps and lengthy processes involved in achieving public reimbursement for treatments like Albrioza in Canada,” Moore said.
“The time is now for equitable and expedited access to approved treatments for ALS, and we will continue advocating on behalf of the ALS community to improve their access to care,” Moore added.
Treatment for ALS facing regulatory hurdles in EU
Albrioza is a fixed-dose oral combination of two small molecules — tauroursodeoxycholic acid and sodium phenylbutyrate — that together are thought to protect nerve cells against forms of cellular stress implicated in ALS. As such, the therapy is expected to slow ALS progression.
Canadian authorities conditionally approved the treatment based on findings from the Phase 2 CENTAUR trial (NCT03127514) and its extension study (NCT03488524). Their findings indicated that Albrioza significantly slowed functional declines and extended survival time for ALS patients.
A full, final approval in the country is contingent upon results from the global Phase 3 PHOENIX trial (NCT05021536), which is now seeking to confirm the benefits of Albrioza against a placebo in a larger group of patients. That trial, which completed enrollment earlier this year, is expected to finish in 2024.
The therapy’s path to approval has not been as straightforward elsewhere.
In the U.S., it came under scrutiny by a U.S. Food and Drug Administration (FDA) advisory committee that initially voted that existing data were not sufficient to support the treatment’s clinical efficacy.
While that opinion is not binding, the FDA takes it into account when deciding about whether to approve a therapy.
Following a rare second review after Amylyx submitted additional trial data, the committee later reversed course. The FDA ultimately approved the therapy, as Relyvrio, in September.
Now, the treatment faces similar hurdles in the European Union, where it’s under review. The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) noted earlier this year that it was leaning toward not recommending the therapy for approval. In March, CHMP sought more information from Amylyx, noting there were still objections to the therapy that needed to be addressed.
As with an FDA advisory committee, the CHMP’s opinion is not binding, but will inform the final decision made by the EMA. The CHMP is expected to give its final opinion in coming days.
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