RARE Daily

Health Canada Approves Amylyx’s ALS Therapy

June 13, 2022

Health Canada has approved Amylyx Pharmaceuticals’ Albrioza for the treatment of amyotrophic lateral sclerosis, the first new therapy approved in Canada for the rare neurodegenerative condition since 2018.

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. Most people with ALS have sporadic disease, showing no clear family history. Approximately 3,000 Canadians are currently living with ALS, and the average life expectancy from symptom onset is two to five years. Approximately 1,000 people die from ALS in Canada every year, with a similar number of diagnoses annually.

Albrioza (sodium phenylbutyrate and taurursodiol) is an oral fixed-dose medication approved with conditions to treat ALS in Canada. The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum (ER) stress and mitochondrial dysfunction. Albrioza is being explored for the potential treatment of other neurodegenerative diseases.

Clinical data demonstrated a statistically significant and clinically meaningful benefit in functional outcomes for people with ALS taking Albrioza compared to people taking placebo, either as a stand-alone therapy or when added to existing treatments for ALS. This decision marks the first marketing approval for Albrioza issued to Amylyx worldwide.

The approval of Albrioza is based on data from CENTAUR, a multicenter phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase, which demonstrated that participants treated with Albrioza scored, on average, 2.32 points higher on the Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) over a period of 24 weeks. Detailed data from CENTAUR were published in the New England Journal of Medicine, Muscle & Nerve in 2020, and the Journal of Neurology, Neurosurgery, and Psychiatry in 2022.

CENTAUR was a multicenter phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. The trial met its primary efficacy endpoint of reducing functional decline as measured by the ALSFRS-R.

Overall, reported rates of adverse events and discontinuations were similar between Albrioza and placebo groups during the 24-week randomized phase; however, GI events occurred with greater frequency (≥2 percent) in the Albrioza (AMX0035) group.  

The CENTAUR trial was funded, in part, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by The ALS Association, ALS Finding a Cure (a program of The Leandro P. Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M. Healey & AMG Center for ALS at Mass General.

The approval of Albrioza was authorized under Health Canada’s Notice of Compliance with Conditions (NOC/c) policy. One of the conditions of the approval is the provision of data from the ongoing phase 3 PHOENIX trial. Other conditions include additional planned or ongoing studies. Amylyx anticipates topline results from the PHOENIX trial in 2024.

“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,” said Tammy Moore, CEO of the ALS Society of Canada. “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.”

AMX0035 has been granted Priority Review by the U.S. Food and Drug Administration  and assigned a Prescription Drug User Fee Act date of September 29, 2022. Additionally, The European Medicines Agency has validated the submission of the company’s Marketing Authorisation application for AMX0035 for the treatment of ALS in Europe and it is under review. A 48-week, randomized placebo-controlled Phase 3 PHOENIX trial of AMX0035 in more than 65 sites across the U.S. and Europe is underway.

“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 (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.”

Author: Rare Daily Staff

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