BMN 250

Drug Profile

BMN 250

Alternative Names: Alpha-N-acetyglucosaminidase-insulin-like-growth-factor-2 fusion protein; BMN250; NAGLU-IGF2 fusion protein

Latest Information Update: 24 Mar 2018

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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

  • 15 Mar 2018 Biomarin Pharmaceuticals plans a phase II trial for Mucopolysaccharidosis III (In children) in Argentina, Australia, Colombia, Germany, Spain, Taiwan, Turkey, United Kingdom and USA (EudraCT-2017-003083-13)
  • 22 Feb 2018 Updated efficacy data from a phase I/II trial in Mucopolysaccharidosis III released by BioMarin Pharmaceutical
  • 06 Sep 2017 Interim efficacy and adverse data from a phase I/II trial in Mucopolysaccharidosis III released by BioMarin Pharmaceutical
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