A new treatment for amyotrophic lateral sclerosis, or ALS, has been approved by the US Food and Drug Administration.
The FDA announced approval of Relyvrio, developed by Amylyx Pharmaceuticals, on Thursday. The oral medication can be taken as a standalone therapy or with other treatments, according to the company, and it has been shown to slow disease progression.
But there’s still some uncertainty about the drug’s efficacy: Amylyx’s submission for approval is based on data from a small Phase 2 trial, and the FDA’s own advisory committee initially voted this spring that the data didn’t show that the drug was effective, before changing its opinion this month. A larger Phase 3 study is still underway.
Source: Relyvrio: ALS drug gets FDA approval despite uncertainty about effectiveness | CNN