FDA Approves Levacetylleucine for Ataxia-Telangiectasia
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The FDA has granted approval to IntraBio's levacetylleucine (Aqneursa), marking the first specific treatment for ataxia in patients with ataxia-telangiectasia.
The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia. The FDA has approved levacetylleucine (Aqneursa; IntraBio) for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing at least 15 kg. The approval marks the first FDA-approved treatment indicated specifically for A-T and is based on data from the phase 3 IB1001-303 trial (NCT06673056). Levacetylleucine previously received approval in September 2024 for neurological manifestations of Niemann-Pick disease type C.
The new indication comes after decades without a therapy approved specifically for A-T, according to the A-T Children's Project. IntraBio has also initiated a phase 3 trial evaluating levacetylleucine in CACNA1A-related disorders, a separate group of rare neurological conditions with no currently approved therapies.
"Today marks a tremendous milestone for the A-T community and IntraBio," Mallory Factor, CEO at IntraBio, said in a statement. "As the first FDA-approved drug for the treatment of ataxia-telangiectasia, AQNEURSA now offers the potential to make meaningful differences in the lives of thousands of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by A-T."
Levacetylleucine efficacy in the IB1001-303 trial
The FDA approval is supported by results from IB1001-303, a randomized, double-blind, placebo-controlled crossover trial conducted at 10 sites across Germany, Slovakia, Spain, Switzerland, the United Kingdom, and the United States. The trial enrolled 73 patients with genetically confirmed A-T, ages 4 to 50 years, and 70 patients (96%) completed the study. Results were published in the July 2026 issue of The Lancet Neurology.
What is levacetylleucine approved for?
Levacetylleucine (AQNEURSA) is approved for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg, in addition to its existing indication for neurological manifestations of Niemann-Pick disease type C.
How does levacetylleucine work?
Levacetylleucine is proposed to normalize glucose metabolism and enhance cerebellar activity, correcting metabolic dysfunction and improving lysosomal and mitochondrial function based on preclinical data.
What did the IB1001-303 trial show?
The phase 3 IB1001-303 trial showed a significant improvement on the SARA ataxia scale with levacetylleucine compared with placebo, with consistent benefit across secondary endpoints and no new safety signals.
