ALS Drug Relyvrio Withdrawn Due to Ineffectiveness

Amylyx Pharmaceuticals has decided to withdraw its ALS drug Relyvrio from the market following disappointing results from a phase III trial. The drug, also known as Albrioza in Canada, is used to treat amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease affecting nerve cells.

The decision to remove Relyvrio was prompted by the trial’s findings that the drug did not perform better than a placebo. Despite being deemed safe, with no new side effects reported, the drug will no longer be available to new patients. However, current patients can continue treatment free of charge.

The drug was granted accelerated approval by the FDA in September 2022, despite initial doubts about its effectiveness. The decision to withdraw it now was based on the PHOENIX trial results, discussions with regulatory authorities, and engagement with the ALS community.

Amylyx Pharmaceuticals will now focus on advancing AMX0035, the drug combination used in Relyvrio, for the treatment of Wolfram syndrome, which causes diabetes and vision loss in children. The company will reduce its workforce by 70% to focus on this and other upcoming clinical milestones.

In 2023, Relyvrio generated $381 million in sales, with $49 million in profits. Currently, only two other drugs, Riluzole and edaravone, have been approved by the FDA for ALS treatment, offering limited options for patients with this devastating disease.