FDA approves Aqneursa for ataxia in ataxia-telangiectasia — first A-T treatment anywhere.

The U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing at least 15 kg (about 33 pounds), according to the FDA human-drugs announcement and IntraBio’s 18 September 2026 Business Wire release, both opened for this pack. Soft FLAG: IntraBio’s “first and only … anywhere in the world” and “commercially available now” lines are company claims via the release; the FDA states this approval makes Aqneursa the first treatment approved for ataxia in patients with A-T.

A-T is a rare inherited neurodegenerative disorder caused by mutations in the ATM gene. It brings progressive loss of muscle control and coordination beginning in early childhood, plus telangiectasias, immune problems and elevated cancer risk. Soft FLAG: IntraBio cites an estimated incidence of about 1 in 40,000 — company epidemiology colour.

The mechanism and label, as sources state them: Aqneursa is a chemically modified amino acid taken as an oral suspension. Soft FLAG: the prescribing information states the distinct molecular target in A-T (and in its prior Niemann-Pick type C indication) is unknown; the proposed mechanism is normalization of glucose metabolism correlated with enhanced cerebellar activity — label/company science, not desk-verified pathway proof. Aqneursa was previously approved in 2024 for neurological manifestations of Niemann-Pick disease type C in patients ≥15 kg; today’s action is a second indication.

Efficacy came from IB1001-303 (NCT06673056), a randomized, double-blind, placebo-controlled, two-period crossover study of 73 patients aged 4 or older with genetically confirmed A-T at sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom and the United States. Seventy patients (96%) completed both periods. Soft FLAG: IntraBio reports a SARA treatment difference of −1.9 points (95% CI −2.7 to −1.1; p<0.001); the FDA primary outcome was the functional SARA (fSARA — gait, sitting, stance, speech), with mean treatment difference −0.6 (95% CI −0.9 to −0.2; p<0.001) — cite fSARA as the agency’s primary. Soft FLAG: company says findings were published in the July 2026 Lancet Neurology — publication cite via company, not a desk re-read of the paper for this pack.

Named actors: Mallory Factor, CEO of IntraBio, called the approval a milestone for the A-T community and said the drug is commercially available now. Brad Margus, founder of the A-T Children’s Project, said families who coped for decades without an A-T-specific approved treatment now have an option that showed benefit across symptoms affecting daily life. Soft FLAG: “thousands of patients and their families” in Factor’s quote is advocacy scale, not a treated-census.

Safety: FDA lists no contraindications but notes possible fetal harm from animal data; most common adverse reactions in A-T were fall, skin laceration and urinary tract infection. IntraBio says no treatment-related serious adverse events or deaths and no discontinuations for treatment-related adverse events in the pivotal study. Avoid concomitant N-acetyl-DL-leucine; monitor P-gp substrates more often. Orphan Drug designation and Priority Review applied to the A-T indication; approval granted to IntraBio Inc.

What to watch without mood: first published U.S. treated-patient or shipment figures after “available now”; any payer coverage criteria tied to fSARA or weight; readout of IntraBio’s ongoing CACNA1A Phase III (separate programme — do not merge into the A-T floor).

Floor: Aqneursa FDA 18 Sep — first ataxia-telangiectasia treatment (FDA/co.); levacetylleucine oral; ≥15 kg; IB1001-303 crossover n=73; fSARA improved vs placebo; available now (co.).