What the FDA decided on Aqneursa for Ataxia-telangiectasia
The FDA approved Aqneursa (levacetylleucine) for ataxia-telangiectasia.
Approved September 18, 2026, 1 day ahead of its action date, as the first treatment for ataxia in ataxia-telangiectasia, for adults and children weighing at least 15kg (about 33 pounds). Aqneursa was already approved in 2024 for Niemann-Pick disease type C. The approval rests on a 73-patient placebo-controlled crossover study (NCT06673056) using the functional SARA ataxia scale; the most common side effects were falls, skin lacerations and urinary tract infections.
IntraBio's own approval release (September 18, 2026) says the drug is commercially available now and is the first approved A-T treatment anywhere in the world.
Who the Aqneursa decision matters to
Ataxia-telangiectasia (A-T) is a rare inherited disorder in which the ATM gene, which coordinates DNA repair, does not work. Children lose coordination from early childhood as the cerebellum degenerates, develop a weakened immune system and dilated blood vessels in the eyes, and carry a sharply raised risk of blood cancers.
On September 18, 2026 the FDA approved Aqneursa (levacetylleucine) to treat the ataxia of A-T, which the FDA called the first treatment approved for ataxia in people with the disease.
Prevalence: 1 in 40,000 to 100,000 people worldwide. See our full Ataxia-Telangiectasia page for current treatments, recruiting trials, and community resources.
How Aqneursa works
Aqneursa: modified amino acid whose exact target is unknown; proposed to normalize glucose and energy metabolism in cerebellar neurons and improve lysosomal and mitochondrial function. Our full Aqneursa profile covers how it works, side effects in plain language, and its trial history.
Where Ataxia-Telangiectasia treatment stands today
Read our full Ataxia-Telangiectasia treatment summary
As of September 18, 2026, A-T has its first approved treatment: Aqneursa (levacetylleucine), an oral suspension approved by the FDA for ataxia in adults and children weighing at least 15kg. It was already on the market for Niemann-Pick disease type C, so it is available now rather than after a launch delay.
In the 73-patient crossover trial that supported approval, ataxia scores improved within 12 weeks on the drug and did not on placebo. It treats the ataxia only; it does not address the immune deficiency, the cancer risk or the lung disease, which continue to need their own management.
Research is active. A large trial of a red-blood-cell-encapsulated steroid (Quince Therapeutics' eDSP) failed in January 2026, and antisense and gene-based approaches are in earlier stages.
The A-T Children's Project funds research, runs a global family data platform, and lists the A-T Clinical Center at Johns Hopkins, the main US referral center. Because of radiation sensitivity, families should ask whether any trial or scan involves radiation exposure, and every A-T patient should be followed for infections and cancer regardless of trial participation.
The Ataxia-Telangiectasia page has every treatment and trial →Meanwhile, 4 Ataxia-Telangiectasia studies are open or about to open
The FDA's decision does not end research on Ataxia-Telangiectasia. Of these, 2 test a treatment; the others follow patients over time. The most recently updated:
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Frequently asked questions
Has the FDA decided on Aqneursa?
The FDA has already decided. It approved the application.
Details: the outcome and what it means are at the top of this page.
Source: FDA approval notice
What was Aqneursa approved for?
The FDA approved IntraBio's sNDA for Aqneursa in Ataxia-telangiectasia.
What to know: Approved September 18, 2026, 1 day ahead of its action date, as the first treatment for ataxia in ataxia-telangiectasia, for adults and children weighing at least 15kg (about 33 pounds). Aqneursa was already approved in 2024 for Niemann-Pick disease type C. The approval rests on a 73-patient placebo-controlled crossover study (NCT06673056) using the functional SARA ataxia scale; the most common side effects were falls, skin lacerations and urinary tract infections. IntraBio's own approval release (September 18, 2026) says the drug is commercially available now and is the first approved A-T treatment anywhere in the world.
How it works: Modified amino acid whose exact target is unknown; proposed to normalize glucose and energy metabolism in cerebellar neurons and improve lysosomal and mitochondrial function.
More: the Aqneursa drug page explains it in plain language.
Is there already an FDA-approved treatment for Ataxia-Telangiectasia?
Yes. Trial Friend's drug list includes 1 FDA-approved drug for Ataxia-Telangiectasia.
Approved: Aqneursa (levacetylleucine).
Check: the Ataxia-Telangiectasia page also searches FDA drug labels for approved treatments.
What did the Aqneursa trials show?
For ataxia-telangiectasia, approval rested on IB1001-303 (NCT06673056), a randomized, double-blind, placebo-controlled crossover trial of 73 patients aged 4 to 50 with genetically confirmed A-T at 10 sites in 6 countries, published in The Lancet Neurology in July 2026. Each patient took levacetylleucine for 12 weeks and placebo for 12 weeks in random order.
More: the Aqneursa drug page has the full trial history and sources.
What are the side effects of Aqneursa?
In the ataxia-telangiectasia trial the most common reactions occurring in at least 5% of patients and more often than on placebo were falls, skin lacerations and urinary tract infection. Falls in a disease defined by loss of balance need care in interpretation: no treatment-related serious adverse events occurred, and no patient stopped because of a treatment-related reaction.
Most common: falls, skin cuts and scrapes, urinary tract infection, abdominal pain (Niemann-Pick C trial), difficulty swallowing (Niemann-Pick C trial), upper respiratory infections (Niemann-Pick C trial).
More: the Aqneursa drug page has the label's warnings in full.
How much will Aqneursa cost?
Trial Friend does not list a price for Aqneursa. What you pay depends on your insurance and on help from the drugmaker or a charity fund.
Drugmaker help: AQNEURSA Cares.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Aqneursa decision?
The FDA approved Aqneursa for Ataxia-telangiectasia on September 18, 2026.
Next steps: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
Where this outcome comes from
The outcome on this page comes from FDA approval notice. This page rechecks against our calendar, which is re-verified daily.