FDA approval expands treatment options for ultra-rare neurologic disease

The FDA approved Aqneursa (levacetylleucine) for ataxia-telangiectasia, positioning it as the first treatment for the disorder. The approval follows an earlier FDA nod for Aqneursa in Niemann-Pick disease type C, extending the company’s chemically modified amino-acid strategy into a new indication with distinct neurodegenerative drivers.