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: 10 Sep 2017

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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

  • 06 Sep 2017 Interim efficacy and adverse data from a phase I/II trial in Mucopolysaccharidosis III released by BioMarin Pharmaceutical
  • 21 Apr 2016 BMN 250 receives Orphan Drug status for Mucopolysaccharidosis III in European Union
  • 22 Mar 2016 Phase-I/II clinical trials in Mucopolysaccharidosis III (In children, In infants) in United Kingdom, Spain (Intracerebral) (NCT02754076) after March 2016
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