Yes. On September 18, 2026, the U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) who weigh at least 15 kg (about 33 pounds). The decision makes Aqneursa the first FDA-approved treatment specifically for ataxia associated with A-T; the drug was previously approved in 2024 for neurological manifestations of Niemann-Pick disease type C.
Approval was granted to IntraBio Inc. and was supported by a randomized, double-blind, placebo-controlled crossover study (NCT06673056) of 73 patients aged 4 years and older. Patients scored better on the functional Scale for the Assessment and Rating of Ataxia (fSARA) while taking Aqneursa than while taking placebo. The agency also granted Orphan Drug designation and Priority Review for the A-T indication.
Source: FDA — Approves Therapy to Treat Ataxia in Patients with Ataxia-Telangiectasia