FDA approval of Relyvrio for the treatment of amyotrophic lateral sclerosis
Amylyx Pharmaceuticals, Inc. announced that the FDA has approved Relyvrio (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS)
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 relentlessly 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.
The approval of Relyvrio is based on data from CENTAUR, a multicenter Phase II clinical trial in 137 participants with ALS encompassing a 6-month randomized, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase. Detailed data from CENTAUR were published in the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry ( previously cited).
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