First FDA-Approved Treatment for Ataxia in Ataxia-Telangiectasia

Aqneursa (levacetylleucine) is a chemically modified amino acid for oral suspension (strawberry-flavored granules in unit-dose packets). On 18 September 2026, the U.S. Food and Drug Administration announced approval of Aqneursa to treat ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg (about 33 pounds). FDA statement: this makes Aqneursa the first treatment approved for ataxia in patients with ataxia-telangiectasia. The same product was previously approved 24 September 2024 for neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg (NDA 219132).

Distinct path — not this label: Supportive physical therapy, speech therapy, infection prophylaxis, or cancer surveillance alone are not this labelled A-T ataxia oral label. Other hereditary ataxias without labelled A-T indication are not this population. The NPC neurological-manifestations label is a separate indicated use of the same molecule — this article is the A-T ataxia pathway. Compounded or over-the-counter leucine products are not this IntraBio NDA label. Today’s approved US oral treatment pathway for A-T ataxia is IntraBio Aqneursa NDA 219132.

Distributed by IntraBio Inc., Austin, TX 78701. Commercial access for eligible patients is organised through AQNEURSA Cares patient support.

Where this is taking place

Approved prescription route today: United States — FDA-labelled Aqneursa for A-T ataxia in adults and pediatrics weighing ≥15 kg, taken as an oral suspension prepared from unit-dose packets (or via qualifying G-tube with water only). Company announcement (18 Sep 2026): commercially available now. Patient navigation / enrollment: AQNEURSA Cares 866-200-0419. Suspected adverse reactions: IntraBio 1-833-306-9677 or FDA MedWatch 1-800-FDA-1088. This is a prescription neurology / genetics conversation — not a DIY supplement and not a DIY import.

FDA Human Drugs news (18 Sep 2026) and IntraBio’s announcement describe the A-T ataxia indication, A-T safety language, and commercial availability. DailyMed SPL retrieved for this article (setid 0f248e55-d1bb-13f9-e063-6294a90a05ce, Revised 9/2024) still listed the NPC indication only. Until the revised SPL posts, treat the FDA announcement + company PI link as the A-T indication source. Ask the prescribing clinician for the current Prescribing Information.

Outside the United States: At the time of writing, no confirmed Health Canada Notice of Compliance / DIN for Aqneursa A-T. Do not import on your own. A US packet is not a foreign pack.

IB1001-303 — NCT06673056 (ClinicalTrials.gov: active but no longer taking new patients) — Pivotal randomized, double-blind, placebo-controlled, two-period crossover Phase 3 study of levacetylleucine in genetically confirmed A-T. FDA description: 73 patients aged 4 years or older (26 adults / 47 pediatric; median age 13, range 4–50); 70/73 (96%) completed; each treatment period 12 weeks. Labelled commercial access is through a prescribing clinician and AQNEURSA Cares. An active (no longer taking new patients) pivotal record is not walk-in research recruitment.

The practical prescription route in the US is a neurology / genetics / ataxia-telangiectasia team confirming ATM genetics, weight ≥15 kg, pregnancy status when relevant, and arranging AQNEURSA Cares / payer logistics.

Approval matrix

Access by country

• United States: Ask a neurology / genetics / A-T clinician about labelled Aqneursa for ataxia. Labelled 18 September 2026. Path: confirm A-T (ATM) → verify weight ≥15 kg → pregnancy testing / contraception when relevant → weight-based packet dosing → AQNEURSA Cares enrollment 866-200-0419. AE: IntraBio 1-833-306-9677. No list price or copay dollars in this article. This is US labelled supply, not a DIY import.

• Canada: Aqneursa A-T authorisation not confirmed. Ask the Canadian A-T / neurology clinic what legal paths exist in Canada when a Canadian A-T label does not yet exist.

• European Union / United Kingdom / Australia / Japan / other countries: Confirm your local regulator’s current status with the treating centre. A US packet is not a foreign pack. Do not import on your own.

• If your regulator has not authorised it: do not import on your own. Ask the local clinician about documented special-access / named-patient rules, referral to a centre in a labelled country, or waiting.

Who is eligible (from FDA / company A-T sources + current USPI dose)

This is an oral labelled expansion conversation — not a cure for all A-T manifestations, and not established below 15 kg.

United States (FDA Human Drugs 18 Sep 2026 + IntraBio announcement + current USPI dose table):

• Indication (A-T): Treatment of ataxia in adult and pediatric patients with ataxia-telangiectasia weighing ≥15 kg.
• Dose (weight-based, 1 g packets): 15 to <25 kg — 1 g morning + 1 g evening (no afternoon dose); 25 to <35 kg — 1 g morning, afternoon, and evening; 35 kg or more — 2 g morning + 1 g afternoon + 1 g evening. Mix each packet in 40 mL water, orange juice, or almond milk (water only for G-tube French size ≥18); swallow within 30 minutes.
• Contraindications: None listed on current USPI / FDA A-T safety summary.
• Warnings: Embryo-fetal toxicity — verify not pregnant before start; effective contraception during treatment and for 7 days after last dose if discontinued.
• Most common A-T adverse reactions (≥5% and greater than placebo): fall, skin laceration, urinary tract infection (FDA / company).
• Drug interactions: Avoid N-acetyl-DL-leucine and N-acetyl-D-leucine; monitor P-gp substrates more frequently.
• Pediatrics: Pivotal included patients from age 4; labelled floor is weight ≥15 kg, not age alone.
• Not a substitute for: immunodeficiency management, infection care, or malignancy surveillance that A-T still requires.

Not labelled on the sources checked for this article:

• Patients weighing <15 kg.
• Canadian (and several other) A-T marketing authorisations (not confirmed here).
• Treating Aqneursa as disease-modifying for every A-T organ system.
• Treating active (no longer taking new patients) research as open commercial trial recruitment.

How to access (United States)

1. Confirm genetically / clinically diagnosed ataxia-telangiectasia with a neurology or genetics clinician who manages A-T.
2. Confirm weight ≥15 kg and discuss pregnancy / contraception when relevant.
3. Ask the clinic to start AQNEURSA Cares (866-200-0419) for benefits investigation, specialty pharmacy routing, and support programs for eligible patients.
4. Learn packet preparation (oral or G-tube) from the Instructions for Use / pharmacist.
5. Plan fall precautions and infection/UTI monitoring given the A-T adverse-reaction profile.
6. Authorised is not funded — insurance, prior authorisation, and out-of-pocket paths vary; no prices in this article.

Evidence snapshot (labelled pivotal)

IB1001-303 — NCT06673056 (FDA Human Drugs + IntraBio; Lancet Neurology July 2026 on company release):

• Design: randomized, double-blind, placebo-controlled, two-period crossover; each period 12 weeks.
• Population: 73 patients with confirmed A-T, aged 4–50 (median 13); 26 adults / 47 pediatric; 38 female / 35 male; 70 (96%) completed.
• Sites (company): 10 centres in Germany, Slovakia, Spain, Switzerland, United Kingdom, and United States.
• FDA primary measure: fSARA (gait, sitting, stance, speech; 0 best → 16 worst) better on Aqneursa than placebo; company-reported fSARA mean treatment difference −0.6 (95% CI −0.9, −0.2; p <0.001); full SARA difference −1.9 (95% CI −2.7, −1.1; p <0.001).
• Safety (company): no treatment-related serious adverse events or deaths; no discontinuations for treatment-related AEs in the reported summary; most common ARs ≥5% and >placebo: fall and skin laceration (FDA also lists urinary tract infection).

CT.gov named status: active but no longer taking new patients (retrieved 21 Sep 2026). Registration fields (estimated enrollment 60, primary completion estimate 2027) may lag the FDA’s completed pivotal description — use the named status and the FDA patient counts for patient navigation honesty.

How to talk with a doctor

Bring this page and ask a neurology / genetics / A-T clinician:

1. “We have genetically confirmed ataxia-telangiectasia and weigh ≥15 kg. Is labelled US Aqneursa for ataxia a fit?”
2. “What is the current AQNEURSA Cares intake step (866-200-0419), and which specialty pharmacy will dispense?”
3. “Which weight-band dose applies, and how should we prepare packets (oral vs G-tube)?”
4. “How will we handle pregnancy testing / contraception, fall risk, and UTI monitoring?”
5. If outside the US: “Has our regulator authorised Aqneursa for A-T, or what legal special-access options exist?”

Who is involved (from primary sources)

• Application holder / distributor: IntraBio Inc., Austin, TX 78701 (FDA announcement; USPI distributor line).
• Patient support: AQNEURSA Cares — 866-200-0419 (IntraBio 18 Sep 2026 announcement).
• Pivotal program: IB1001-303 NCT06673056.
• AE contact: IntraBio 1-833-306-9677; FDA MedWatch 1-800-FDA-1088.
• Advocacy cited on company release: A-T Children’s Project (founder Brad Margus quoted) — advocacy contact is not a prescribing path.

Names and roles above are limited to what appears on the primary sources cited. Contacts not listed on those sources are omitted.

Sources

• FDA Human Drugs: “FDA Approves Therapy to Treat Ataxia in Patients with Ataxia-Telangiectasia…”, 18 Sep 2026
• IntraBio Business Wire / investor release, 18 Sep 2026 (indication language, IB1001-303 results, AQNEURSA Cares phone, commercial availability)
• DailyMed / USPI — AQNEURSA (Rev 9/2024; NDA 219132; NDC 83853-101-01) — dose, NPC history, pregnancy warning, interactions (A-T SPL posting gap flagged)
• ClinicalTrials.gov NCT06673056 (active but no longer taking new patients)
• FDA NPC approval history (24 Sep 2024) for prior labelled use of the same NDA

Authorized is not funded. Not medical advice.