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Tralesinidase alfa - BioMarin Pharmaceutical

Drug Profile

Tralesinidase alfa - BioMarin Pharmaceutical

Alternative Names: Alpha-N-acetyglucosaminidase-insulin-like-growth-factor-2 fusion protein; AX-250; BMN 250; NAGLU-IGF2 fusion protein

Latest Information Update: 05 Nov 2023

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At a glance

  • Originator BioMarin Pharmaceutical
  • Class Acetylglucosaminidases; Recombinant fusion proteins
  • Mechanism of Action Alpha N acetyl D glucosaminidase replacements
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 09 Aug 2022 Allievex Corporation plans a phase IIIb/IV trial for Mucopolysaccharidosis III (In children, In adults, In the elderly) in the USA, Colombia, Germany, Turkey and the United Kingdom (Intracerebroventricular, Infusion) in September 2022 (NCT05492799)
  • 23 Jul 2020 Allievex Corporation completes the phase I/II trial in Mucopolysaccharidosis III (In Infants, In children) in Germany, Spain, Taiwan, UK, Turkey, Colombia and USA (Intracerebral) (NCT02754076)
  • 23 Oct 2019 Tralesinidase alfa to be licensed to Allievex worldwide
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