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Levacetylleucine - IntraBio

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Drug Profile

Levacetylleucine - IntraBio

Alternative Names: AQNEURSA; IB-1001 - IntraBio; N-Acetyl-L-Leucine - IntraBio

Latest Information Update: 30 Oct 2025

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At a glance

  • Originator University of Munich; University of Oxford
  • Developer IntraBio
  • Class Acetamides; Amino acids; Esters; Neuroprotectants; Pentanoic acids; Small molecules; Vestibular disorder therapies
  • Mechanism of Action Calcium channel modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Tay-Sachs disease; Niemann-Pick disease type C; Ataxia telangiectasia
  • New Molecular Entity Yes

Highest Development Phases

  • Registered Niemann-Pick disease type C
  • Phase III Ataxia telangiectasia
  • Phase II Sandhoff disease; Tay-Sachs disease

Most Recent Events

  • 27 Oct 2025 IntraBio plans a phase III trial for Spinocerebellar ataxias, Ataxia, Migraine-with-aura (In children, In adolescents, In adults, In the elderly) in Germany, Switzerland and USA (PO) (NCT07221292)
  • 29 Jul 2025 Preregistration for Niemann-Pick disease type C (In adolescents, In children, In the elderly, In adults) in European Union (PO) before July 2025
  • 29 Jul 2025 European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommends approval of Levacetylleucine for Niemann-Pick disease type C in European Union

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