FDA Approves Amylyx Drug to Treat ALS

Cambridge-based Amylyx Pharmaceuticals has won FDA approval for Relyvrio for the treatment of amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig’s disease. Relyvrio significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled clinical trial. Relyvrio can be taken as a monotherapy or with existing approved treatments.

ALS is a progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S.

Relyvrio, a combination of sodium phenylbutyrate and taurursodiol, is an oral fixed-dose medication with marketing applications also pending in the EU and approved with conditions to treat ALS in Canada. The combination of sodium phenylbutyrate and taurursodiol may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction. It is also being explored for the potential treatment of other neurodegenerative diseases. (Source: Amylyx Pharmaceuticals Website, 07 and 29 September, 2022)