FDA Approves Aqneursa for Ataxia-Telangiectasia

The U.S. FDA has approved Aqneursa® (levacetylleucine) for the treatment of ataxia in adults and children with ataxia-telangiectasia (AT) weighing at least 15 kg, providing a new treatment option for this rare genetic disorder.

AQNEURSA is the first and only drug approved for the treatment of A-T anywhere in the world. In the United States, it is the second indication AQNEURSA is approved to treat, following its initial approval in September 2024 for the treatment of the neurological manifestations in Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

“Today marks a tremendous milestone for the A-T community and IntraBio. 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,” said Mallory Factor, Chief Executive Officer of IntraBio. “This approval is the result of years of work by investigators, advocacy organizations, and above all the patients and families who took part in this research. We are proud to bring the first approved treatment to the A-T community, and we are committed to making sure that families who could benefit from AQNEURSA are able to access it.”

A-T is a rare, progressive, and prematurely fatal autosomal-recessive neurodegenerative disorder that affects an estimated 1 in 40,000 people. A-T is characterized by progressive cerebellar degeneration resulting in worsening ataxia that can affect gait, truncal control, eye movements, balance, speech, and hand coordination, alongside other systemic manifestations. Until today, there were no approved treatments for A-T anywhere in the world.

“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. We are grateful to every family who participated in this research and to IntraBio for their commitment to a disease this rare and this complex. We will keep working to make sure every patient who could benefit has access to it."

PIVOTAL PHASE 3 RESULTS

The approval is supported by the pivotal IB1001-303 study (NCT06673056), a randomized, double-blind, placebo-controlled, crossover trial that enrolled 73 patients aged 4 to 50 years with genetically confirmed A-T across 10 clinical trial sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom, and the United States.

The trial met the primary efficacy endpoint and key secondary endpoints were supportive of the primary result. Results from the study showed AQNEURSA significantly improved signs and symptoms of ataxia and demonstrated functional benefits important to everyday life that were evident within 12 weeks. Seventy patients (96%) completed the study and received both placebo and AQNEURSA. These findings were published in the July 2026 issue of The Lancet Neurology.

Key findings included:

• AQNEURSA demonstrated statistically significant improvement compared to placebo on the primary efficacy endpoint, the Scale for the Assessment and Rating of Ataxia (SARA) with a treatment difference of -1.9 points (95% CI: –2.7, –1.1; two-sided p<0.001).

• The primary outcome assessed by the FDA was the functional SARA (fSARA), a modified version comprising the gait, sitting, stance, and speech disturbance domains. Patients who received AQNEURSA showed a greater improvement in fSARA score, with a mean treatment difference of −0.6 (95% CI: −0.9, −0.2; two-sided p<0.001).

• Improvement was consistent across secondary endpoints and prespecified subgroups, including pediatric and adult patients, with no evidence of heterogeneity in treatment effect across the population.

• AQNEURSA was well tolerated. No treatment-related serious adverse events and no deaths occurred, and no patients discontinued because of a treatment-related adverse event. The most common adverse reactions (incidence ≥5% and greater than placebo) were fall and skin laceration.