ALS drug funded by Ice Bucket Challenge finally wins FDA approval

Cambridge, Massachusetts based Amylyx Pharmaceuticals has received FDA approval for ALS aka Lou Gehrig's disease drug "Relyvrio". All Things Considered has an excellent article on the controversy surrounding FDA's recent Relyvrio approval:

Proponents of the drug say the small trial showed that it works. But FDA scientists and an expert panel that advises the FDA, weren't so sure. Typically, FDA approval requires two independent studies – each with hundreds of participants – showing effectiveness, or one large study with clearly positive results. In March, the Peripheral and Central Nervous System Drugs Advisory committee concluded that the Amylyx study did not provide "substantial evidence" that its drug was effective. Then in September, during a rare second meeting to consider a drug, the panel reversed course and voted in favor of approval.

All Things Considered | NPR