.Pharmacolibrary.Drugs.ATC.A.A16AB09 .Pharmacolibrary.Drugs.ATC.A.A16AB09| name: | Idursulfase |
| ATC code: | A16AB09 | route: | intravenous |
| n-compartments | 2 |
Idursulfase is a recombinant enzyme replacement therapy used for the treatment of Hunter syndrome (mucopolysaccharidosis II), a rare lysosomal storage disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. The drug is approved for use in patients with this genetic condition and is administered via intravenous infusion.
Pharmacokinetic parameters based on data from male pediatric and adult patients with Hunter syndrome (MPS II) receiving 0.5 mg/kg intravenous infusions of idursulfase once weekly.
Giugliani, R, et al., & Pano, A (2014). A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy. Genetics in medicine : official journal of the American College of Medical Genetics 16(6) 435–441. DOI:10.1038/gim.2013.162 PUBMED:https://pubmed.ncbi.nlm.nih.gov/24202085