Idursulfasa
Idursulfasa Uses, Dosage, Side Effects, Food Interaction and all others data.
Idursulfasa is a purified form of human iduronate-2-sulfatase, a lysosomal enzyme. Idursulfasa is produced by recombinant DNA technology in a human cell line. Idursulfasa is an enzyme that hydrolyzes the 2-sulfate esters of terminal iduronate sulfate residues from the glycosaminoglycans dermatan sulfate and heparan sulfate in the lysosomes of various cell types. Idursulfasa is a 525-amino acid glycoprotein with a molecular weight of approximately 76 kilodaltons. The enzyme contains eight asparagine-linked glycosylation sites occupied by complex oligosaccharide structures. The enzyme activity of idursulfase is dependent on the post-translational modification of a specific cysteine to formylglycine.
Idursulfasa is a purified form of the lysosomal enzyme human iduronate-2-sulfatase of recombinant DNA origin. It is designed to replace the natural enzyme, increasing catabolism of certain accumulated glycosaminoglycans (GAG), which abnormally accumulate in multiple tissue types in patients with mucopolysaccharidosis II (MPS-II, or Hunter syndrome).
| Trade Name | Idursulfasa |
| Availability | Prescription only |
| Generic | Idursulfase |
| Idursulfase Other Names | Idursulfasa, Idursulfase |
| Related Drugs | Elaprase |
| Type | |
| Formula | C2654H4000N688O774S14 |
| Weight | 76000.0 Da |
| Groups | Approved |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Idursulfasa is a purified lysosomal enzyme used as a replacement therapy for Hunter syndrome in pediatric and adult patients.
For the treatment of Hunter syndrome in adults and children ages 5 and older.
Idursulfasa is also used to associated treatment for these conditions: Mucopolysaccharidosis II
How Idursulfasa works
Hunter's Syndrome is an X-linked recessive disease caused by insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. This enzyme cleaves the terminal 2-O-sulfate moieties from the glycosaminoglycans (GAG) dermatan sulfate and heparan sulfate. Due to the missing or defective iduronate-2-sulfatase enzyme in patients with Hunter's Syndrome, GAG progressively accumulate in the lysosomes of a variety of cells, leading to cellular engorgement, organomegaly, tissue destruction and organ system dysfunction. Treatment of Hunter's Syndrome patients with idursulfase provides exogenous enzyme for uptake into cellular lysosomes. Targeting of idursulfase to the lysosome occurs by endocytosis from the cell surface. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow specific binding of the enzymes to the M6P receptors on the cell surface, leading to cellular internalization of the enzyme, targeting to intracellular lysosomes and subsequent catabolism of accumulated GAG.
Toxicity
There is no experience with overdosage of Idursulfasa in humans. Single intravenous doses of idursulfase up to 20 mg/kg were not lethal in male rats and cynomolgus monkeys (approximately 6.5 and 13 times, respectively, of the recommended human dose based on body surface area) and there were no clinical signs of toxicity.
Food Interaction
No interactions found.Idursulfasa Disease Interaction
Half Life
44 ± 19 minutes
Clearance
- 3 mL/min/kg [Patients (7.7 – 27 years) with Hunter syndrome with treatment week 1(0.5 mg/kg ELAPRASE administered weekly as a 3-hour infusion)]
- 3.4 mL/min/kg [patients (7.7 – 27 years) with Hunter syndrome with treatment week 27 (0.5 mg/kg ELAPRASE administered weekly as a 3-hour infusion)]
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