Cambridge: Amylyx Pharmaceuticals, Inc. has announced that Health Canada has approved ALBRIOZA (sodium phenylbutyrate and ursodoxicoltaurine), with conditions, for the treatment of amyotrophic lateral sclerosis (ALS). Clinical data demonstrated a statistically significant and clinically meaningful benefit in functional outcomes for people with ALS taking ALBRIOZA (also known as AMX0035) 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.
“For nearly a decade, we have been committed to creating more meaningful moments for people living with ALS and their families. We are excited with Health Canada’s decision to approve ALBRIOZA with conditions. ALBRIOZA is a therapy that demonstrated in our CENTAUR trial a statistically significant and clinically meaningful impact on function, alone or in addition to existing ALS therapies. We are grateful to the people who participated in our clinical trials, their families, the researchers, and the ALS community for helping to make this milestone happen,” said Justin Klee and Joshua Cohen, Co-CEOs and Co-Founders of Amylyx.
ALS is a relentlessly progressive and fatal neurodegenerative 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. The vast majority of people with ALS (>90%) 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.
“I have always had a love for life and a passion for global advocacy. Thanks to new innovations in ALS treatment and care, I’ve been able to continue my advocacy work in new ways as part of the fight for new treatment options for the ALS community,” said Norman MacIsaac, who has been living with ALS since 2014. “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.”
“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, FRCP(C), 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.”
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.
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, and the Journal of Neurology, Neurosurgery, and Psychiatry.
“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. 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.”
“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.”