Drug Profile


Alternative Names: Aldurazyme; Alpha-L-iduronidase; BM 101; JC 0498; rh alpha-L-iduronidase

Latest Information Update: 08 Nov 2017

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At a glance

  • Originator BioMarin Pharmaceutical; Genzyme Corporation
  • Developer BioMarin Pharmaceutical; Sanofi Genzyme; University of California at Los Angeles
  • Class Glycoside hydrolases
  • Mechanism of Action Iduronidase replacements
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis I
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Mucopolysaccharidosis I

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for phase-I development in Mucopolysaccharidosis-I(In children) in USA (Intrathecal, Injection)
  • 08 Aug 2017 Launched for Mucopolysaccharidosis I in Russia and Hong Kong (IV) before August 2017
  • 15 Feb 2017 Interim pharmacodynamics data from a trial in mucopolysaccharidosis type I (Hurler syndrome) released by Sanofi Genzyme
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