FDA Approves IntraBio’s Aqneursa as First Treatment for Ataxia-Telangiectasia

Editor’s Take: This FDA approval addresses a long-awaited treatment option for patients living with A-T.

The U.S. FDA approved the IntraBio drug Aqneursa (levacetylleucine) to treat ataxia in adult and pediatric patients with ataxia-telangiectasia (A-T). This is the first and only approved treatment for A-T anywhere in the world, IntraBio says.

It also marks Aqneursa’s second FDA approval, following an indication for neurological manifestations in Niemann-Pick disease type C (NPC). That came in September 2024.

Aqneursa Impact on A-T, NPC

A-T is a rare disease affecting an estimated 1 in 40,000 people. It is a progressive, prematurely fatal autosomal-recessive neurodegenerative disorder. A primary A-T characteristic, progressive cerebellar degeneration, results in worsening ataxia. This affects gait, truncal control, eye movements, balance, speech, hand coordination, and other systemic manifestations.

NPC, meanwhile, is a slowly progressive lysosomal disorder, for which principal manifestations are age dependent. Infants who survive may have delayed psychomotor development; some others may present with neurologic disease years later.

“Today marks a tremendous milestone for the A-T community and IntraBio,” company CEO Mallory Factor said. “Aqneursa now offers the potential to make meaningful differences in the lives of thousands of patients and their families … This approval is the result of years of work by investigators, advocacy organizations, and above all the patients and families.”

IntraBio Earns High FDA Marks

IntraBio says the FDA’s approval for the A-T indication stemmed from a pivotal Phase III study. This trial enrolled 73 patients, ages 4 to 50, in the United States, United Kingdom, Germany, Slovakia, Spain, and Switzerland.

According to IntraBio, Aqneursa significantly improved signs and symptoms of ataxia in these patients. Furthermore, the drug demonstrated functional benefits key to everyday life, evident within 12 weeks.