Amylyx Pharmaceuticals Announces FDA Approval of Relyvrio for the Treatment of ALS

The U.S. Food and Drug Administration approved Amylyx Pharmaceuticals’ Relyvrio, an oral combination therapy for the rare, neurodegenerative disease amyotrophic lateral sclerosis.

Photo: Sabrina Paganoni, principal investigator of the CENTAUR trial

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 percent 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 was based on a multicenter phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label extension long-term follow-up phase. The trial met its primary efficacy endpoint.

“Any time we have a new tool to slow the progression of this disease represents an important milestone in how we battle ALS,” said Sabrina Paganoni, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and associate professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital. “The published data on both function and survival in a randomized trial—and what this means for people living with ALS—are a step forward for the ALS community.”

The most common adverse events occurring with Relyvrio (at least 15 percent and at least 5 percent greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first three weeks of treatment.

In an investor call after the approval, Amylyx said it will price Relyvrio around $158,000, a price it said was below the latest FDA-approved product available to people with ALS and following the input of many stakeholders throughout the ALS community in the United States. It said it did that to ensure that every eligible person who can benefit from Relyvrio will have access.