Canada Approves Amylyx's ALS Drug; FDA To Consider Approval In September

The Canadian government has given its approval to AMX0035, a new drug from Cambridge, Massachusetts-based Amylyx Pharmaceuticals Inc. AMLX for the treatment of amyotrophic lateral sclerosis (ALS), the progressive neurodegenerative disease also known as Lou Gehrig’s disease.

What Happened: Health Canada’s approval of the drug is the first federal regulatory approval for Amylyx and the first new ALS therapy approved in Canada since 2018.

According to Amylx, the new drug – which the company markets under the brand name Albrioza – is a combination of two existing drugs, sodium phenylbutyrate and taurursodiol. Health Canada’s approval followed Amylyx’s Phase 2 study of 137 patients, which found those receiving the drug over six months recording a 25% slower decline on average than those who received placebos.

The company also noted that one of the conditions of Health Canada’s approval is the provision of data from the ongoing Phase 3 trial. Amylyx anticipated topline results from the Phase 3 trial in 2024.

“The approval by Health Canada, the first for Amylyx globally, is an important milestone and first step,” said Chris Aiello, general manager and head of Canada at Amylyx. “We anticipate that Albrioza will be available commercially in Canada within the next six weeks. We are prepared to work with the pan-Canadian Pharmaceutical Alliance 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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What Happens Next: According to the Wall Street Journal, the U.S. Food and Drug Administration (FDA) is scheduled to announce its approval decision for the drug by Sept. 29. The initial deadline for the decision was extended by three months after the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 in determining the Phase 2 trial did not adequately demonstrate its effectiveness.

However, the FDA can override the advisory committee’s opinion and approve the drug.

In Canada, approximately 3,000 people have been diagnosed with ALS, which impacts nerve cells in the brain and spinal cord. According to the ALS Association, in roughly 90% of all cases, there is no known family history of the disease or genetic mutation linked to ALS.

“ALS is a devastating disease and can move with startling swiftness,” said Tammy Moore, CEO of the ALS Society of Canada. “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. 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. There is simply no time to wait with this disease.”

Photo: Michal Jarmoluk / Pixabay