Time Matters in A-T. Treatment is here.

AQNEURSA is the first and only FDA-approved treatment for Ataxia-Telangiectasia (A-T)¹

In a Phase 3 multinational, randomized, double-blind, placebo-controlled trial, AQNEURSA demonstrated statistically significant and clinically meaningful improvements in signs and symptoms of ataxia in adult and pediatric patients with A-T.^1,2

Indication

AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg

About A-T (Ataxia-Telangiectasia)

A-T is a relentlessly progressive, fatal disease that places a substantial burden on patients

A rare, inherited neurodegenerative disease caused by mutations in the ATM gene, with an estimated incidence of 1 in 40,000 to 100,000 births^2,3
Characterized by progressive cerebellar ataxia, immunodeficiency, and a predisposition to cancer⁴
Progressive ataxia typically begins in early childhood and can increasingly affect gait, balance, speech, swallowing, and fine motor control⁴

AQNEURSA was studied in a Phase 3 pivotal trial to address the urgent need for a treatment for ataxia in A-T^1,2

About AQNEURSA

AQNEURSA is the first and only FDA-approved treatment for ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.¹

Efficacy

In A-T, function lost is rarely regained. In the pivotal trial, AQNEURSA significantly improved signs and symptoms of ataxia and functional outcomes vs placebo.

The efficacy of AQNEURSA was established in a Phase III randomized, double-blind, placebo-controlled crossover trial in 73 adults and children aged 4 to 50 years weighing ≥15 kg with genetically confirmed A-T.^1,2

AQNEURSA significantly improved signs and symptoms of ataxia and functional outcomes vs placebo as measured by the primary endpoint fSARA (treatment difference −0.6; 95% CI: −0.9, −0.2; p<0.001) and as supported by the SARA (−1.9; 95% CI: −2.7, −1.1; p<0.001).^1,2*

In both treatment sequences, improvement occurred while patients were receiving AQNEURSA - not while they were receiving placebo.^1,2

Full Trial Design and Results

Safety

AQNEURSA has a well-established safety and tolerability profile in patients with A-T.

The most common adverse reactions (incidence ≥5% and greater than placebo) were fall, skin laceration, and urinary tract infection.¹

No treatment-related serious adverse events or deaths occurred, and no adverse events led to discontinuation of study drug.²

The most common adverse reactions (incidence ≥5% and greater than placebo) were fall and skin laceration.¹ No treatment-related serious adverse events or deaths occurred, and no adverse events led to discontinuation of study drug.²

Dosing and Administration

AQNEURSA features oral dosing and administration that are designed to fit into patients’ daily routines.

To access AQNEURSA, download and complete the Start AQNEURSA Prescription Form.

*The SARA and fSARA are clinical assessments of ataxia. The SARA scores 8 domains for a total of 0 (best neurological status) to 40 (worst). The fSARA is a modified version scoring the gait, sitting, stance, and speech disturbance domains, for a total of 0 (best) to 16 (worst). Lower scores indicate better neurological status, so a negative treatment difference favors AQNEURSA.¹ Mean (SD) SARA total score was 19.8 (6.4) with AQNEURSA and 19.9 (6.4) with placebo at baseline, and 18.0 (7.3) and 19.7 (7.4), respectively, at the end of the 12-week treatment period. Mean (SD) fSARA total score was 7.8 (2.6) with AQNEURSA and 7.9 (2.6) with placebo at baseline, and 7.2 (2.8) and 7.8 (3.0), respectively, at the end of the 12-week treatment period.²

Helpful Resources

A-T Organizations

A-T Children’s Project

IntraBio is not responsible for the content or services of this organization. Inclusion of this link on this website is not intended as an endorsement of the organization or the services offered.

Downloadable Resources

Resources to help access an AQNEURSA prescription:

AQNEURSA Prescription Enrollment Form

Prior Authorization Checklist

A-T Sample Letter of Medical Necessity

A-T Sample Letter of Appeal

Resources with more information about AQNEURSA:

A-T Patient Brochure

Full Prescribing Information

Instructions for Use

A-T HCP Brochure

AQNEURSA Cares program

The AQNEURSA Cares program can help eligible patients access treatment.

Learn more by downloading the brochure that's right for you:

AQNEURSA Cares Patient Brochure

AQNEURSA Cares HCP Brochure

Indication

AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of ataxia-telangiectasia (A-T) in adults and children weighing ≥15 kg.

Important Safety Information

Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.

Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall and skin laceration.

These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please click here for Full Prescribing Information for AQNEURSA.

References:

AQNEURSA. Prescribing information. IntraBio.
Martakis K, et al. Lancet Neurol. 2026 Jul;25(7):633-644.
Crawford TO. Semin Pediatr Neurol. 1998;5(4):287-294.
Rothblum-Oviatt C, et al. Orphanet J Rare Dis. 2016;11(1):159.

Important Safety Information

Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

Indication

AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.

Please click here for Full Prescribing Information for AQNEURSA.

References:
1. AQNEURSA. Prescribing information. IntraBio.
2. Martakis K, et al. Lancet Neurol. 2026 Jul;25(7):633-644.
3. Crawford TO. Semin Pediatr Neurol. 1998;5(4):287-294.
4. Rothblum-Oviatt C, et al. Orphanet J Rare Dis. 2016;11(1):159.

Important Safety Information

Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

Indication

AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.

Please click here for Full Prescribing Information for AQNEURSA.

Time Matters in A-T. Treatment is here.

AQNEURSA is the first and only FDA-approved treatment for Ataxia-Telangiectasia (A-T)1

About A-T (Ataxia-Telangiectasia)

About AQNEURSA

Helpful Resources

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Important Safety Information

Indication

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AQNEURSA is the first and only FDA-approved treatment for Ataxia-Telangiectasia (A-T)¹