Hunter Syndrome (Mucopolysaccharidosis Type II) Medication

Updated: Apr 18, 2018
  • Author: Germaine L Defendi, MD, MS, FAAP; Chief Editor: Maria Descartes, MD  more...
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Medication Summary

Idursulfase, a purified form of human iduronate sulfatase (IDS) was approved by the US Food and Drug Administration (FDA) as an orphan drug in July 2006. It is distributed as Elaprase (Shire Human Genetics Therapies, Inc). FDA approval was based on the study of 96 patients in a double-blind, placebo-controlled study over one year. [39, 40] This study demonstrated improvement in a 6-minute walk test and reduction in liver and spleen volumes and urinary glycosaminoglycan (GAG) levels.

The extent to which enzyme replacement therapy (ERT) delays disease progression and whether or not it can prevent premature death is still unknown. Severely affected patients were not enrolled, and thus the benefit to them remains to be determined. ERT does not enter or affect the CNS and hence has no effect on cognitive function. Thus, the role of ERT in the management of Hunter syndrome is under debate. [41]



Class Summary

ERT is a life-long therapy that may improve the quality of life for patients with mucopolysaccharidosis type II (MPS II).

Idursulfase (Elaprase)

Purified form of human iduronate-2-sulfatase, a lysosomal enzyme. Hydrolyzes 2-sulfate esters of terminal IDS residues from the GAGs dermatan sulfate and heparan sulfate in the lysosomes of various cell types. Indicated for MPS II (Hunter syndrome) because it replaces the deficiency of iduronate-2-sulfatase in this disease. The drug is continued throughout life, and, thus, both the time and financial commitment can be extensive. Administration should be done by a health care professional in an experienced infusion center.