FDA approves Aqneursa, first treatment for ataxia in ataxia-telangiectasia

FDA has approved Aqneursa (levacetylleucine) to treat ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing at least 15 kg.

A-T is a rare, inherited neurodegenerative disorder that typically begins in early childhood. Progressive loss of muscle control and coordination can impair walking, balance, and speech. There is no cure, and treatment options for neurological symptoms have been limited. Ongoing care must also address the immune deficiencies and increased cancer risk associated with A-T, alongside management of neurological manifestations.

Aqneursa is a modified amino acid whose specific molecular target in A-T is unknown. The oral suspension is taken two or three times daily, depending on body weight. It was previously approved in 2024 for neurological manifestations of Niemann-Pick disease type C.

Approval for ataxia in patients with A-T was supported by a randomized, double-blind, placebo-controlled phase 3 trial involving 73 patients aged 4 to 50 years. Patients received Aqneursa and placebo in successive 12-week periods, with treatment order randomly assigned. Ataxia scores were better during Aqneursa treatment.

Results were measured using the functional Scale for Assessment and Rating of Ataxia, which evaluates walking, standing, sitting, and speech. On this scale, which ranges from 0 to 16, lower scores indicate less severe impairment. Adjusted average scores were 7.2 with Aqneursa versus 7.8 with placebo, a statistically significant 0.6-point difference favoring treatment.

The most common adverse reactions included falls, skin lacerations, and urinary tract infections. Labeling warns of potential fetal harm based on animal studies. Pregnancy should be excluded before treatment, and patients who could become pregnant should use effective contraception during treatment and for seven days after the last dose.

“This is a historic day for the A-T community and the patients and caregivers who have coped for decades without a treatment approved specifically for A-T,” said Brad Margus, founder of the A-T Children’s Project. “With today’s FDA decision, those impacted by A-T have a treatment option that has demonstrated benefit across a range of A-T symptoms, including those that affect daily life for patients and their loved ones.”

Sources: FDA. (2026 Sep 18). FDA Approves Therapy to Treat Ataxia in Patients with Ataxia-Telangiectasia, a Rare Genetic Disorder; IntraBio. (2026 Sep 18). IntraBio Announces U.S. FDA Approval of AQNEURSA® (levacetylleucine) for Ataxia-Telangiectasia