FDA Approves AQNEURSA for Ataxia-Telangiectasia — the First Drug Ever Approved for A-T Anywhere in the World
On September 18, 2026, IntraBio announced FDA approval of AQNEURSA (levacetylleucine) for the treatment of ataxia in adult and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg. Per the company: "AQNEURSA is the first and only drug approved for the treatment of A-T anywhere in the world.12" Until now there were no approved treatments for A-T in any country. It is also AQNEURSA's second U.S. indication, following its September 2024 approval for the neurological manifestations of Niemann-Pick disease type C. (Business Wire)
A-T is a progressively fatal autosomal-recessive neurodegenerative disorder (ATM gene, ~1 in 40,000 people) causing cerebellar degeneration, immunodeficiency, and markedly elevated cancer risk. Brad Margus, founder of the A-T Children's Project: "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."
The evidence base
Approval rests on the pivotal IB1001-303 study (NCT06673056) — a randomized, double-blind, placebo-controlled crossover trial of 73 patients aged 4–50 with genetically confirmed A-T across 10 sites in Germany, Slovakia, Spain, Switzerland, the UK, and the US; 96% completed both arms. Results were published in the July 2026 issue of The Lancet Neurology:
- Primary endpoint (SARA ataxia scale): treatment difference −1.9 points (95% CI −2.7 to −1.1; p<0.001); the FDA-designated functional SARA (gait, sitting, stance, speech) showed −0.6 (p<0.001).
- Benefits were evident within 12 weeks and consistent across pediatric and adult subgroups.
- Well tolerated: no treatment-related serious adverse events and no deaths; most common adverse reactions were fall, skin laceration, and urinary tract infection.
AQNEURSA is a first-in-class chemically modified amino acid taken as an oral suspension; its molecular target in A-T and NPC is unknown, with a proposed mechanism of normalizing glucose metabolism correlated with enhanced cerebellar activity. IntraBio is also running Phase 3 (IB1001-304) in CACNA1A-related disorders, another condition with no approved therapies.
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An instance of First-in-history drug approvals now arrive in waves, not once per generation. — A uniformly fatal neurodegenerative disease with zero approved treatments anywhere gets its first drug inside the same season's wave of first-ever clearances. ↩︎
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An instance of The absence of approved treatments for a rare disease escalates pipeline assets into high-stakes acquisition and registration races. — In a fatal disease that never had an approved therapy anywhere, securing the first-ever worldwide registration is the definitive first-mover prize the absence of competitors escalates. ↩︎