Elaprase
Elaprase is a recombinant DNA-derived form of the human enzyme iduronate-2-sulfatase. It is used as an enzyme replacement therapy for patients with Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II). Elaprase is produced by Shire plc and was approved by the Food and Drug Administration (FDA) in 2006.
Medical Uses[edit | edit source]
Elaprase is indicated for the treatment of Hunter syndrome, a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This enzyme deficiency leads to the accumulation of glycosaminoglycans (GAGs) in various tissues and organs, causing a wide range of symptoms including developmental delays, organomegaly, and skeletal abnormalities.
Mechanism of Action[edit | edit source]
Elaprase works by replacing the deficient or absent iduronate-2-sulfatase enzyme in patients with Hunter syndrome. This enzyme is essential for the breakdown of GAGs, specifically dermatan sulfate and heparan sulfate. By providing the functional enzyme, Elaprase helps reduce the accumulation of GAGs, thereby alleviating some of the symptoms associated with the disorder.
Administration[edit | edit source]
Elaprase is administered via intravenous infusion. The recommended dosage is 0.5 mg/kg of body weight, given once weekly. The infusion typically lasts for 3 to 4 hours and should be administered under the supervision of a healthcare professional.
Side Effects[edit | edit source]
Common side effects of Elaprase include headache, fever, rash, and infusion-related reactions such as flushing, chills, and hypotension. Severe allergic reactions, including anaphylaxis, have also been reported. Patients should be monitored for signs of hypersensitivity during and after the infusion.
Regulatory Status[edit | edit source]
Elaprase received orphan drug designation from the FDA and the European Medicines Agency (EMA) due to the rarity of Hunter syndrome. It was approved by the FDA in July 2006 and by the EMA in January 2007.
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