Almost 80% of novel agent submissions from the biggest companies were approved in recent years, compared with a 60% approval rate for the rest of the industry, according to a Pink Sheet analysis of FDA approval decisions since 2021.

US FDA designates ongoing ATLAS trial in presymptomatic individuals with SOD1 mutation as confirmatory study. ‘Classic Biogen’ approval is first in ALS under the expedited pathway and could lead to more drugs reaching market on basis of a reduction in plasma neurofilament light chain concentration.

Pink Sheet reporters and editors discuss the FDA’s upcoming advisory committee meeting for the controversial ALS cell therapy NurOwn, a proposal to batch test all drugs coming into the US amid problems conducting foreign inspections, and the FDA’s return to in-person meetings with industry.

A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.