FDA Approves IntraBio’s Aqneursa for Ataxia-Telangiectasia

Rare Daily Staff

The U.S. Food and Drug Administration has approved IntraBio’s Aqneursa to treat ataxia in people with ataxia-telangiectasia, marking the first approved treatment for the rare neurological disease.

The oral medicine is approved for adults and children who weigh at least 33 pounds. It is commercially available now.

Ataxia-telangiectasia, often called A-T, is a rare genetic disorder that typically begins in early childhood and progressively affects the brain and other body systems. It is caused by disease-causing changes in both copies of the ATM gene. Damage to the cerebellum, a part of the brain responsible for coordination and balance, leads to worsening ataxia, including difficulty walking, sitting upright, speaking clearly, controlling eye movements and using the hands. People with A-T may also develop immune-system problems, recurrent lung and sinus infections, difficulty swallowing and a substantially increased risk of cancer, particularly blood cancers. The condition can shorten life expectancy.

Aqneursa is a chemically modified amino acid taken by mouth as a liquid suspension. Its precise molecular target is not known. IntraBio says the drug is designed to address metabolic problems in cells, including abnormalities involving lysosomes and mitochondria, potentially improving cerebellar activity.

Aqneursa was previously approved in September 2024 for neurological symptoms of Niemann-Pick disease type C, another rare inherited disorder. The new clearance is its second FDA-approved use.

“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,” Brad Margus, founder of the A-T Children’s Project, said in IntraBio’s announcement.

The approval was based on a randomized, double-blind, placebo-controlled crossover trial known as IB1001-303. The study enrolled 73 people ages 4 to 50 with genetically confirmed A-T at 10 sites in the United States and five European countries.

Participants received both Aqneursa and placebo during different periods of the study. Seventy participants, or 96 percent, completed the trial and received both treatments.

After 12 weeks, Aqneursa improved patients’ scores on the Scale for the Assessment and Rating of Ataxia, or SARA, compared with placebo. The average treatment difference was 1.9 points in favor of Aqneursa. On a modified functional version of the scale focused on gait, sitting, stance and speech, the average difference was 0.6 points in favor of the drug.

The company said benefits were consistent among children and adults and across other prespecified subgroups. Results from the trial were published in the July 2026 issue of The Lancet Neurology, according to the release.

No treatment-related serious adverse events, deaths or treatment discontinuations were reported in the A-T trial, IntraBio said. The most common adverse reactions occurring more often with Aqneursa than with placebo were falls, skin lacerations and urinary tract infections. The drug carries warnings related to pregnancy.

Photo: Brad Margus, founder of the A-T Children’s Project