Amylyx meeting helped clarify regulatory flexibility, says a proponent of the concept

Patients have been calling for it. FDA has promised it. But what regulatory flexibility means for ALS and other neurological diseases, and where its limits lie, have remained opaque. Last week’s advisory committee meeting to discuss Amylyx’s ALS therapy shed some light on the concept.

FDA's Office of Neuroscience Director Billy Dunn kicked off the Sept. 7 meeting by stating that there should be no mystery about the degree of flexibility afforded to FDA under the law or about the agency’s imperative to apply “maximum” flexibility for severe diseases with substantial unmet need...