Idursulfase in Hunter syndrome treatment

Drugs Today (Barc). 2007 Nov;43(11):759-67. doi: 10.1358/dot.2007.43.11.1157619.

Abstract

Hunter syndrome (mucopolysaccharidosis II, MPS II) is a rare X-linked lysosomal storage disorder caused by the deficiency of enzyme iduronate-2-sulfatase (I2S), which results in accumulation of undegraded dermatan and heparan sulfate in various tissues and organs. Enzyme replacement therapy with Elaprase (idursulfase, a recently approved orphan product) is the first treatment for Hunter syndrome. Results of the randomized, double-blind, placebo-controlled phase II/III clinical trial of idursulfase demonstrated that weekly infusions of idursulfase increase walking distance and improve pulmonary function as well as reduce organ size and urinary glycosaminoglycans (GAGs) excretion in MPS II patients. Idursulfase is generally well tolerated, although infusion reactions do occur. Clinical studies demonstrate that idursulfase may be the first successful symptomatic therapy that can benefit patients with MPS II by addressing the enzymatic defect.

Publication types

  • Review

MeSH terms

  • Animals
  • Controlled Clinical Trials as Topic
  • Drug Evaluation, Preclinical
  • Female
  • Glycoproteins / deficiency
  • Humans
  • Iduronate Sulfatase / adverse effects
  • Iduronate Sulfatase / pharmacokinetics
  • Iduronate Sulfatase / pharmacology*
  • Male
  • Mucopolysaccharidosis II / drug therapy*
  • Mucopolysaccharidosis II / enzymology
  • Mucopolysaccharidosis II / genetics

Substances

  • Glycoproteins
  • IDS protein, human
  • Iduronate Sulfatase
  • idursulfase