Topline
Drug manufacturer Amylyx Pharmaceuticals announced on Thursday plans to remove Relyvrio from the market after lackluster results from a phase III trial, leaving only two other ALS drugs on the market.
Key Facts
Relyvrio (also known as Albrioza in Canada) is used to treat amyotrophic lateral sclerosis (ALS), a fatal and progressive neurodegenerative disease that affects the brain and nerve cells in the spinal cord.
The process to remove the drug from the market in both Canada and the U.S. will begin Thursday, and it will no longer be available to new patients, though current patients who wish to continue taking the drug can do so free of charge, according to the company’s announcement.
The results of the 48-week phase III trial involving 664 participants were announced in March, and the researchers concluded the drug didn’t have better results than the placebo, though the researchers still deemed it safe since no new side effects occurred.
After the results of the trial were announced, watchdog group Public Citizen urged the drug manufacturer to “promptly” remove Relyvrio from the market; the group previously asked the Food and Drug Administration to not grant approval for the drug in 2022 because its phase II trial results were “inconclusive.”
Relyvrio was granted accelerated approval by the FDA in September 2022 after an FDA advisory committee initially said in the spring of 2022 its phase II trial results didn’t prove the drug was effective, though it changed its tune later that year.
The advisory committee wrote in a summary following the approval that “uncertainty about the evidence of effectiveness” still remained, but it proceeded with its approval because ALS is life-threatening and serious, so “this level of uncertainty is acceptable in this instance.”
Crucial Quote
“The decision to remove Relyvrio/Albrioza from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community,” Joshua Cohen and Justin Klee, co-CEOs of Amylyx Pharmaceuticals, said in a statement.
What To Watch For
Relyvrio is the brand name for sodium phenylbutyrate and taurursodiol, or AMX0035. Amylyx Pharmaceuticals stated in its Thursday announcement it would continue advancing AMX0035 for treatment in Wolfram syndrome, which typically causes diabetes and vision loss in children. The results of its phase II trial will be announced on April 10 during a company webcast. The company will reduce its workforce by 70% to focus on this and other upcoming clinical milestones.
Big Number
$381 million. That’s how much Relyvrio generated in sales in 2023, according to Amylyx Pharmaceuticals’ full year financial report. The drug made $49 million in profits that same year.
Key Background
Only two other ALS drugs have been granted FDA approval. Riluzole was approved in 1995, and works by blocking the release of glutamate, a neurotransmitter that’s a part of the nervous system, according to the University of Michigan. Too much glutamate can potentially damage nerve cells. according to the National Library of Medicine’s MedlinePlus. It typically extends life expectancy by two to three months, and increases the chances of another year of survival by about 9%. The second medication called edaravone was approved in 2017, and works by slowing down nerve damage caused by worsening ALS symptoms. It slows progression by around 33%, which may be equivalent to four to five additional months of survival in comparison to riluzole, according to a 2020 study published in the Canadian Medical Association Journal.
Further Reading
ALS drug fails large clinical trial and may be withdrawn from market (CNN)
