Amylyx’s Relyvrio receives FDA approval for ALS treatment

Amylyx’s Relyvrio (sodium phenylbutyrate and taurursodiol) has been approved by the US Food and Drug Administration (FDA) for the treatment of adults with amyotrophic lateral sclerosis (ALS), the company announced.

The FDA’s decision is based on positive results from a phase 2 trial in which Relyvrio (previously known as AMX0035 in the US) was shown to significantly slow the loss of physical function in people living with ALS.

Characterised by a progressive degeneration of motor nerve cells in the brain and spinal cord, ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually death.

The phase 2 CENTAUR trial enrolled 137 participants with ALS, encompassing a six-month, randomised, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase.

Detailed data from the study was published in the New England Journal of Medicine, Muscle & Nerve and the Journal of Neurology, Neurosurgery, and Psychiatry.

Joshua Cohen and Justin Klee, co-chief executive officers of Amylyx said the approval was “an exciting milestone for the ALS community”.

They added: "We want to give a heartfelt thank you to the broader ALS community, including healthcare professionals and those living with ALS, for their guidance, support of our clinical programmes and for sharing their experiences with us. Their stories inspired us and helped our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning and there is much more to be done.”

The approval follows previous concerns from the FDA advisory committee that resulted in a 6-4 negative vote for the drug in March this year.

The European Medicines Agency is reviewing the company’s marketing authorisation application for Relyvrio for this same indication in Europe, Amylyx repored.

Margaret Olinger, global head of commercial and chief commercial officer of Amylyx, said: “Our priority now is to ensure that adults living with ALS in the US whose doctors have prescribed Relyvrio can access it as quickly as possible.

“Physicians will be able to prescribe immediately, and we anticipate specialty pharmacies will be able to start to fill prescriptions and ship Relyvrio to people with ALS in the next four to six weeks.”