Amylyx Pharmaceuticals (Nasdaq:AML) has won FDA approval for Relyvrio (AMX0035; sodium phenylbutyrate and ursodoxicoltaurine), notching the first win for amyotrophic lateral sclerosis (ALS) in five years.
The drug received Health Canada approval in June. The drug is known as Albrioza in that country.
In a summary, FDA reviewers note that the limited clinical data available for the drug have resulted in “in a degree of residual uncertainty about the evidence of effectiveness” of the drug. The agency notes that the lack of clarity is acceptable given the significant disease burden of ALS.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Dr. Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a news release. “The FDA remains committed to facilitating the development of additional ALS treatments.”