Two FDA-linked rare-disease milestones landed this week, underscoring regulators’ growing comfort with expedited pathways in high-unmet-need areas. IntraBio’s Aqneursa (levacetylleucine) received an FDA nod as the first treatment for ataxia-telangiectasia, following its earlier approval in Niemann-Pick disease type C. The update positions a single development program across related lysosomal and neurodegenerative indications. Elsewhere, Otsuka and Ionis Pharmaceuticals’ ALS candidate is now set to move into regulator discussions after hitting a primary endpoint in a Phase 3 study. Otsuka is reported to be heading to the FDA, with the partners positioned to seek expedited approval routes tied to ALS’s severity and limited treatment options. Taken together, these events show a regulatory pattern: when trials deliver clear clinical signals in genetically defined or otherwise high-need populations, companies are increasingly able to compress the time between pivotal readouts and agency engagement.