Amid FDA delay, Amylyx scores conditional ALS drug nod in Canada

Amylyx Pharmaceuticals may be navigating a tortuous regulatory path in the U.S. for its amyotrophic lateral sclerosis drug, but it just scored a major win north of the border. 

Health Canada granted conditional approval to the med, AMX0035, under the brand name Albrioza. It's the company's first approval anywhere in the world—and it comes after some tough setbacks in the U.S.

After a downbeat analysis from the FDA's own internal reviewers, an independent FDA advisory panel recently voted down the ALS therapy, saying it needed more trial data to prove itself. The U.S. agency delayed its decision deadline until Sept. 29. 

Health Canada approved Albrioza based on data from a phase 2 trial called CENTAUR and a longer open-label follow-up. For the CENTAUR trial, investigators tested Albrioza against placebo for six months in 137 patients with ALS. After 24 weeks, patients on the drug scored 2.32 points higher—on average—on the Amyotrophic Lateral Sclerosis Functional Rating Scale.  

After seeing those data, FDA staffers earlier this year said the study was “not exceptionally persuasive.” They said the company’s analysis should’ve included deaths that occurred in the natural course of the disease even though those deaths were not a result of the treatment.   

Under the Canadian conditional approval, Amylyx will have to turn in data from its ongoing Phase 3 PHOENIX trial, which is expected to read out in 2024. Canadian regulators are also requiring “additional planned or ongoing studies,” Amylyx said in its approval announcement. 

Albrioza becomes the first ALS drug approved in Canada since Mitsubishi Tanabe’s approval for Radicava back in October 2018. Before that, ALS patients had relied on just one drug—riluzole—for two decades. 

ALS is a severe neurodegenerative condition that is usually fatal two to five years after diagnosis. 

"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," Tammy Moore, CEO of the ALS Society of Canada, said in a statement. "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.”

While Amylyx celebrates the win in Canada, the biotech no doubt has larger markets in mind. U.S. reviewers are set to decide on the medicine this fall, and a review is also underway in Europe. 

Elsewhere in the ALS treatment field, Mitsubishi Tanabe recently won FDA approval for the oral version of its own drug, which can be taken at home and, if necessary, through a feeding tube. Prior to that approval, the treatment required hourlong infusions at clinics 10 days a month. Amylyx's drug is also an oral fixed-dose medicine.