FDA approves the first drug for ataxia in ataxia-telangiectasia

The US Food and Drug Administration approved Aqneursa (levacetylleucine) oral suspension from IntraBio on 18 September 2026 for the treatment of ataxia in adults and children with ataxia-telangiectasia who weigh at least 15 kg. The agency describes it as the first treatment approved for ataxia in the disease.

Ataxia-telangiectasia is a rare inherited disorder in which the cerebellum degenerates from early childhood, taking with it the coordination needed to walk, sit steadily and speak. The approval rests on a randomised, double-blind, placebo-controlled crossover study (NCT06673056) of 73 patients, 47 of them children, with a median age of 13 and a range of 4 to 50. Patients on the drug scored better than on placebo on the functional Scale for the Assessment and Rating of Ataxia, a 0-to-16 measure covering gait, sitting, stance and speech.

The drug carried orphan designation and priority review. It is the second indication for Aqneursa, which the FDA first approved in 2024 for the neurological manifestations of Niemann-Pick disease type C.