HEALTH
ALS Drug Pulled From Market After It Fails To Show Benefit In Large Study
Amylyx Pharmaceuticals
The maker of a drug for ALS, also known as Lou Gehrig’s disease, is pulling it from the market after a large study found it showed no benefit for patients.
Amylyx Pharmaceuticals on Thursday announced it begun a process with the U.S. Food and Drug Administration and Health Canada to end the marketing and sales of the drug, sodium phenylbutyrate–taurursodiol, known as Relyvrio in the U.S. and Albrioza in Canada.
As of Thursday, the drug will no longer be available to new patients, the company said. Those currently taking the medication who wish to continue the treatment, in consultation with their physician, can be transitioned to a free drug program.
The move comes after the results of the Phase 3 PHOENIX trial of the drug indicated it didn’t help patients with ALS, or amyotrophic lateral sclerosis, a progressive neurodegenerative disease that may begin with muscle twitching and weakness before eventually progressing to death.
“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases, Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, said in a statement. 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. Thank you to each and every person who shared feedback with us and continues to support our commitment to the ALS community.
The drug continues to be tested for the treatment of Wolfram syndrome, an inherited condition that typically causes diabetes and vision loss, and progressive supranuclear palsy (PSP), a rare brain condition that impairs movement, including eye function.
The drug was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum stress and mitochondrial dysfunction, said Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx, and those two pathways are also implicated in Wolfram syndrome and PSP.
For ALS, the company says another drug in its pipeline shows strong potential for treating the disease.
We also remain focused on ALS and believe AMX0114 has strong potential for the treatment of ALS and other diseases, Bedrosian said. In our preclinical studies of AMX0114 and in multiple independent published studies, inhibition of calpain-2 has reduced cell death and degeneration and decreased neurofilament levels. We expect to initiate a clinical trial studying AMX0114 in ALS in the second half of this year.”
With the failure of Relyvrio to treat ALS, Amylyx announced it will be cutting its workforce by 70% to refocus its efforts on other clinical trials.
“We are so thankful and grateful to our Amylyx team for their contributions and steadfast dedication,” said Cohen and Klee. “Together, the work we have accomplished across the world has helped build a vital foundation to achieve our mission of one day ending the suffering caused by neurodegenerative diseases, which continue to have critical, unaddressed needs.”
TMX contributed to this article.
