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Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Company-reported prevalence

Aqneursa

Overview

FieldValue
DrugAqneursa (levacetylleucine)
IndicationAtaxia-telangiectasia
DiseaseAtaxia-Telangiectasia (A-T)
CompanyIntraBio Inc. (worldwide rights; no partner holding rights identified)
Documents searchedIntraBio corporate website: home, pipeline, lead program, products, clinical trials, news list, and ataxia-telangiectasia disease pages, accessed October 2026; AQNEURSA product website: ataxia-telangiectasia page and patient pages, accessed October 2026; AQNEURSA healthcare professional website, accessed October 2026; press release, January 21, 2026 (pivotal trial topline results); press release, January 2026 (European Commission approval in Niemann-Pick disease type C); press release, February 11, 2026 (further pivotal trial detail); press release, March 20, 2026 (sNDA submission); press release, May 19, 2026 (sNDA acceptance and priority review); press release, June 2026 (EMA variation application); press release, September 2026 (FDA approval); securities filings: none found (privately held company); corporate or investor presentation: none found
Data as ofOctober 14, 2026
Prepared byAI research agent (Claude); not yet reviewed by a person

Company statements of prevalence

United States

Number diagnosed: IntraBio press release, February 2026

“Public reports estimate there are approximately 4,600 diagnosed A-T patients in the U.S.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) press release, February 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Europe

Number affected: IntraBio press release, February 2026

“about 5,000 A-T patients in the U.K. and EU4 countries.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) press release, February 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Global

Prevalence: IntraBio website, October 2026

“A-T is a rare inherited disorder that affects an estimated 1:40,000 – 100,000 people worldwide.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) ataxia-telangiectasia disease page, quoted passage highlighted (opens the full-size image in a new tab)
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Other countries and regions

No evidence found.

Geography not stated

Incidence: IntraBio AQNEURSA website, October 2026

“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” (opens the source at this quote in a new tab)

Cited source: 2.1 Martakis 2026; 2.2 Crawford 1998

IntraBio (2026) AQNEURSA ataxia-telangiectasia website, quoted passage highlighted (opens the full-size image in a new tab)
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Prevalence: IntraBio press release, September 2026

“A-T is a rare, progressive, and prematurely fatal autosomal-recessive neurodegenerative disorder that affects an estimated 1 in 40,000 people.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) press release, September 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Prevalence: IntraBio press release, June 2026

“A-T is estimated to affect 1 in 40,000-100,000 people.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) press release, June 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Prevalence: IntraBio press release, January 2026

“Regulatory submissions planned in the US, Europe, and other jurisdictions for levacetylleucine in Ataxia-Telangiectasia, a neurodegenerative disease without any approved treatment and impacting approximately 1 in 70,000 people” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

IntraBio (2026) press release, January 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Sources the company cites

Martakis 2026

Reference as the company lists it

“Martakis K, et al. Lancet Neurol. 2026 Jul;25(7):633-644.” (opens the source at this quote in a new tab)

IntraBio (2026) AQNEURSA ataxia-telangiectasia website, quoted passage highlighted (opens the full-size image in a new tab)
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Passage carrying the figure

“Ataxia-telangiectasia is a rare (incidence one in 40 000–100 000 people), progressive, debilitating, and prematurely fatal autosomal-recessive inherited cerebellar ataxia.” (opens the source at this quote in a new tab)

Martakis 2026, quoted passage highlighted (opens the full-size image in a new tab)
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Crawford 1998

Reference as the company lists it

“Crawford TO. Semin Pediatr Neurol. 1998;5(4):287-294.” (opens the source at this quote in a new tab)

IntraBio (2026) AQNEURSA ataxia-telangiectasia website, quoted passage highlighted (opens the full-size image in a new tab)
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Passage carrying the figure

Not quoted: Closed access (Seminars in Pediatric Neurology, Elsevier); the PubMed abstract does not state the figure.

References

  1. Crawford 1998 (opens in a new tab)
  2. IntraBio (2026) AQNEURSA ataxia-telangiectasia website (opens in a new tab)
  3. IntraBio (2026) ataxia-telangiectasia disease page (opens in a new tab)
  4. IntraBio (2026) press release, February 2026 (opens in a new tab)
  5. IntraBio (2026) press release, January 2026 (opens in a new tab)
  6. IntraBio (2026) press release, June 2026 (opens in a new tab)
  7. IntraBio (2026) press release, September 2026 (opens in a new tab)
  8. Martakis 2026 (opens in a new tab)