First-Ever Approval for Treatment of Rare Neurologic Disease A-T…IntraBio’s Aqneursa

IntraBio’s neurologic drug Aqneursa has received the world’s first approval as a treatment for ataxia-telangiectasia (A-T), a rare neurodegenerative disease.

According to Fierce Pharma on the 22nd (local time), the U.S. Food and Drug Administration (FDA) expanded Aqneursa’s indication and approved it to treat patients with A-T. A-T is a rare neurodegenerative disease that causes premature death.

The approval was based on results from a phase 3 clinical trial (IB1001-303) announced earlier this year. In a crossover study involving 73 patients with A-T ages 4 to 50, Aqneursa significantly improved ataxia symptoms compared with placebo within 12 weeks of treatment.

Scores on the Scale for the Assessment and Rating of Ataxia (SARA) improved by about 2 points. On the functional SARA score, which focuses on walking, sitting, standing and speech and was prioritized by the FDA, Aqneursa showed a 0.6-point advantage. There were no serious adverse events, and no patients discontinued treatment because of adverse events.

Brad Margus, founder of the A-T Children’s Project, said, “This is a historic day for patients and caregivers who have endured for decades without a treatment specifically for A-T.”

A-T is caused by mutations in the ATM gene and leads to the progressive loss of balance, coordination and fine motor skills. It is also associated with immunodeficiency, speech impairment and an increased risk of cancer, and typically appears in early childhood. Its prevalence worldwide is estimated at about 1 in 40,000 people.

The FDA approved Aqneursa oral suspension for all pediatric and adult patients with A-T who weigh at least 15 kg.

Mallory Factor, IntraBio’s chief executive officer (CEO), said, “We will ensure that families who could benefit from Aqneursa can access the drug.” The company operates a financial assistance program to help reduce out-of-pocket costs of $15,000 every 1–2 weeks.

A-T is Aqneursa’s second indication. The drug was approved by the FDA in 2024 to treat Niemann-Pick disease type C, a lysosomal storage disorder, and received the same approval from the European Commission (EC) in early 2026.

How Aqneursa treats the two neurologic diseases is not yet clear. IntraBio believes it works by correcting the brain’s energy metabolism rather than treating a specific genetic defect.

Privately held IntraBio is testing this theory in a third indication. It is nearing completion of enrollment in a phase 3 trial for a group of disorders caused by CACNA1A gene mutations. According to the company, about 30,000 people in the United States alone have CACNA1A disorders, and, as with A-T, there are no approved treatments. IntraBio plans to exceed its enrollment target and begin data collection by October.