Albrioza, Oral ALS Therapy Known as AMX0035, Approved in Canada

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Albrioza, formerly called AMX0035, has been given conditional approval by Health Canada for the treatment of amyotrophic lateral sclerosis (ALS).

The decision marks a first approval for Albrioza by a regulatory authority in any country and the first new ALS therapy to enter the Canadian market since 2018. Amylyx Pharmaceuticals, the company developing the medication, had applied for Albrioza’s approval in Canada in mid-2021.

“The approval by Health Canada, the first for Amylyx globally, is an important milestone and first step,” Chris Aiello, general manager and head of Canada at Amylyx, said in a press release.

“We anticipate that Albrioza will be available commercially in Canada within the next six weeks,” Aiello added. “We are prepared to work with the pan-Canadian Pharmaceutical Alliance (pCPA) and federal, provincial and territorial governments to negotiate the listing of Albrioza expeditiously, so that eligible Canadians living with ALS can obtain access as quickly and efficiently as possible.”

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The approval came under Health Canada’s notice of compliance with conditions policy, which enables therapies showing the potential to fill an unmet medical need in severe, life-threatening diseases to reach the market sooner, provided that certain conditions are met.

One such condition is the provision of data from the ongoing Phase 3 clinical trial called PHOENIX (NCT05021536) that is investigating Albrioza’s safety and effectiveness in approximately 600 ALS patients. The trial, with enrolled sites in the U.S., is or should soon be recruiting participants at dozens of locations across Europe. Results are expected in 2024.

Other planned or ongoing studies are also part of the conditional approval.

“We are delighted that this Health Canada approval represents the advancement in available treatment options for people living with ALS, but there is still more work to be done,” said Tammy Moore, CEO of the ALS Society of Canada.

“ALS is a devastating disease and can move with startling swiftness. It is incredibly important that all Canadians across the country are able to benefit from these and other innovations to come, as quickly as possible following regulatory approval,” Moore said. “We are hopeful that decision-makers throughout the drug access and reimbursement process will work expeditiously to provide timely and equitable access for all Canadians who may benefit.”

Albrioza is an oral medicine containing a fixed dose of two small molecules — tauroursodeoxycholic acid and sodium phenylbutyrate — that are believed to help protect nerve cells from stress and damage.

Health Canada’s approval was supported by data from the Phase 2 CENTAUR clinical trial (NCT03127514), which enrolled 137 adults with rapidly progressing ALS. Participants were randomly assigned to Albrioza, then known as AMX0035, or to a placebo, taken twice daily for six months. Most patients entered into its extension study (NCT03488524) where all were given the medication.

Top-line results from CENTAUR showed that treatment with Albrioza significantly slowed functional decline compared with a placebo. Trial analyses also suggested that the treatment would extend median survival times by more than 10 months if placebo-assigned patients in the main study had stayed on placebo, instead of switching to active treatment during its open-label extension. Treatment also prolonged the time until patients required a tracheostomy or ventilation by over seven months.

“New therapies like Albrioza are critical to help me and others have a fuller life. The ability to live more independently for longer is incredibly meaningful,” said Norman MacIsaac, who has been living with ALS since 2014.

“For more than 30 years, I have been involved with caring for people living with ALS, and we have had major breakthroughs in research and made strides in drug development. The approval of Albrioza is a testament to this progress,” said Angela Genge, MD, director of the ALS Global Centre for Excellence at the Montreal Neurological Institute.

“New treatment options that can slow disease progression are critical to give people living with ALS more time as we work toward a cure,” Genge added.

Albrioza is up for approval in the U.S., with a decision expected in September. A committee of the U.S. Food and Drug Administration recently voted narrowly against recommending approval, saying that there is not yet enough evidence to prove the medicine is effective. The therapy also is being considered for approval in Europe.