FDA Approves Aqneursa, First Ataxia Treatment for Ataxia-Telangiectasia

The FDA approved levacetylleucine (Aqneursa, IntraBio) oral suspension to treat ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) who weigh at least 15 kg. The agency said the decision makes Aqneursa the first approved treatment for ataxia in patients with A-T. The drug was previously approved in 2024 for neurological manifestations of Niemann-Pick disease type C.

A-T is a rare, inherited neurodegenerative disorder caused by mutations in the ATM gene. It primarily affects the nervous system, causing progressive loss of muscle control and coordination that typically begins in early childhood. The disorder is also associated with telangiectasias, immune deficiencies, and an elevated cancer risk.

Efficacy was evaluated in a randomized, double-blind, placebo-controlled, 2-period crossover trial involving 73 patients aged 4 to 50 years with confirmed A-T. Participants received Aqneursa and placebo in randomly assigned sequences, with each treatment period lasting 12 weeks. The population included 26 adults and 47 pediatric patients; 70 participants, or 96%, completed the study.

The FDA assessed efficacy using the functional Scale for Assessment and Rating of Ataxia (fSARA), which evaluates gait, sitting, stance, and speech disturbance on a 0-to-16 scale, with lower scores indicating better neurological status. Patients had better fSARA scores while receiving Aqneursa than while receiving placebo. IntraBio reported a mean treatment difference of −0.6 points (95% CI, −0.9 to −0.2; 2-sided P<.001).

The most common adverse reactions in patients with A-T were falls, skin lacerations, and urinary tract infections. Aqneursa may cause fetal harm based on animal data. Concomitant use with N-acetyl-DL-leucine should be avoided, and patients receiving P-glycoprotein substrates should be monitored more frequently for related adverse reactions.

Aqneursa received Orphan Drug designation and Priority Review for the A-T indication. Approval was granted to IntraBio.

Source: US Food and Drug Administration. FDA approves therapy to treat ataxia in patients with ataxia-telangiectasia, a rare genetic disorder. Accessed September 20, 2026.

IntraBio Inc. IntraBio announces US FDA approval of Aqneursa (levacetylleucine) for ataxia-telangiectasia. Press release. IntraBio Inc.; September 18, 2026. Accessed September 20, 2026.