FDA approves oral, fixed-dose therapeutic for adults with ALS

The FDA has approved Relyvrio, an oral, fixed-dose combination therapy for the treatment of adults with ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

According to the release, Relyvrio (sodium phenylbutyrate and taurursodiol, Amylyx) significantly slowed the loss of physical function in people with ALS in a randomized, placebo-controlled clinical trial, results of which have been published in several medical journals. ALS currently affects approximately 29,000 Americans, the release stated, and more than 90% of adults with ALS have sporadic disease, with no family history.

“The approval of Relyvrio represents a positive step forward for the ALS community, as they now have a new treatment option in the fight against this relentless and universally fatal disease," Amylyx Co-CEOs Joshua Cohen and Justin Klee told Healio. "ALS treatment has been historically slow and riddled with failures, making timely access to safe and effective treatments especially important. With [this] approval, Relyvrio is the first drug to show a benefit on both function and survival, and the data has been published widely in peer-reviewed medical journals."

The approval of Relyvrio is based on data from a multicenter, phase 2 clinical trial in 137 participants with ALS, which included a 6-month randomized, placebo-controlled phase, followed by an open-label extension, long-term phase. The most common adverse events reported were diarrhea, abdominal pain, nausea and upper respiratory tract infection, the release stated. Gastrointestinal-related adverse events occurred throughout the study but were more common in the first 3 weeks.

“The ALS community continues to inspire and challenge us to help our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward," Cohen and Klee said.