Health Canada gave Albrioza the green light on a conditional basis.Sean Kilpatrick/The Canadian Press
Health Canada has become the first regulator in the world to approve a new amyotrophic lateral sclerosis (ALS) drug, only the third therapy for treatment of the fatal motor neuron condition to be authorized in 20 years.
Amylyx Pharmaceuticals, based in Massachusetts, announced the decision Monday, saying the approval was made with the condition the company will provide Health Canada with further evidence of the drug’s safety and efficacy once a larger trial is completed in 2024.
ALS patients and researchers celebrated the news, highlighting how few treatment options exist for those living with the disease, which progressively robs people of their ability to move, speak, swallow and breathe.
“Anything that can slow the progression of this disease and allow people to retain their function longer is so significant,” said Tammy Moore, chief executive officer of the ALS Society of Canada.
First person: Living with the uncertainty of ALS has taught me some valuable lessons
Justin Klee, co-founder and co-chief executive officer of Amylyx, said in an interview that this drug approval in Canada will bring hope to patients around the world.
“I think it’s an enormous milestone, particularly for the ALS community in Canada, but I think more broadly globally as well,” he said.
About 1,000 Canadians are diagnosed with ALS each year and another 1,000 die of the disease annually. 80 per cent of those with ALS die within two to five years of diagnosis. The new drug, Albrioza, is a combination of sodium phenylbutyrate and ursodoxicoltaurine. In a clinical trial, it appeared to help slow the rate of decline in people who took it compared with those who received a placebo.
Despite the enthusiasm from patient groups, there’s been some debate as to whether the clinical results justify approval.
The decision to authorize Albrioza in Canada was made months after an advisory panel to the U.S. Food and Drug Administration said there was not enough evidence the drug is effective and therefore should not be approved. The FDA was set to make an announcement on the drug by June 29, but recently announced it would delay the decision until late September.
While Health Canada gave Albrioza the green light, it did so on a conditional basis, provided the company provide supplemental data on safety and efficacy after the completion of larger clinical studies. The conditional approval, which is often done to allow patients access to potentially promising drugs, could be revoked if the larger trial doesn’t show a clear benefit.
Ottawa neurologist Ari Breiner said he and many of his colleagues believe the available data demonstrate a benefit and that while they await more clinical results, they are supportive of the drug’s authorization. Dr. Breiner, who was not involved with the study, noted that so far, research has shown the drug produced about a 25-per-cent reduction in functional rating scores. Supplemental research showed that patients taking the drug lived, on average, 6.5 months longer.
“Certainly it’s something that we’re going to consider for patients,” said Dr. Breiner, clinical research chair in ALS at the Éric Poulin Centre for Neuromuscular Disease at the University of Ottawa. “Saying to someone, is six months extra with your family valuable? I think the answer is yes.”
Angela Genge, medical director of the Clinical Research Unit at the Montreal Neurological Institute, said what some are interpreting as a modest benefit is actually significant when it comes to ALS. Few drugs exist to slow progression of the disease and Albrioza appears to do just that, allowing some patients to maintain certain abilities for several additional months. Dr. Genge said she’s seen instances where drugs to treat more common diseases, such as cancer, are approved even though the evidence of benefit is slim.
“The result is clinically significant,” said Dr. Genge, who has received honoraria for work on Amylyx advisory boards. “So I believe giving a [notice of compliance with conditions] is the right way to go.”
Norman MacIsaac, who was diagnosed with ALS in 2014, has been taking Albrioza under Health Canada’s Special Access Programme and said he has noticed his fine motor skills have improved, making it easier for him to pick up pills on his own instead of having to rely on a caregiver. Mr. MacIsaac, who has been one of only a few individuals with ALS who survive five years after diagnosis, said the drug gives him hope of more time.
“For me, the drug comes with the promise of breathing on my own for longer,” Mr. MacIsaac said in a recent interview.
Ms. Moore of the ALS Society said the next hurdle will be securing approval from provincial drug plans to fund Albrioza so that it’s accessible to patients. This can often be a time-consuming process, which presents a challenge.
Editor’s note: June 27th, 2022: The estimated completion date of the Amylyx Pharmaceuticals trial is 2024; a previous version of this story incorrectly said it would be this summer.
Our Morning Update and Evening Update newsletters are written by Globe editors, giving you a concise summary of the day’s most important headlines. Sign up today.