How do you administer Elaprase?
Elaprase is given as an infusion into a vein. A healthcare provider will give you this injection, usually once per week. Elaprase must be given slowly through an IV infusion, and the infusion can take up to 3 hours or longer to complete.
How is Elaprase made?
ELAPRASE is a formulation of idursulfase, a purified form of human iduronate-2-sulfatase, a lysosomal enzyme. Idursulfase is produced by recombinant DNA technology in a human cell line.
How is Idursulfase produced?
Idursulfase is produced by recombinant DNA technology in a human cell line. Idursulfase 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.
How much does elaprase cost?
Shire Pharmaceuticals’ Elaprase ($375,000 per year) treats an ultra-rare metabolic disorder called Hunter’s syndrome.
How much does ELAPRASE cost?
Does ELAPRASE have a black box warning?
Black Box Warning RISK OF ANAPHYLAXIS: Life-threatening anaphylactic reactions have occurred in some patients during and up to 24 hours after ELAPRASE infusions.
How common is Hunter’s syndrome?
How common is Hunter syndrome? Hunter syndrome is almost always diagnosed in males. Doctors diagnose it in roughly 1 out of 100,000 to 170,000 males. Females can be carriers of the genetic mutation that causes MPS II.
Which is the expensive drug in the world?
The most recent treatment is Zolgensma (generic name onasemnogene abeparvovec), a pioneering gene therapy dubbed “the most expensive drug in the world” and only available through the NHS since March 2021. Zolgensma uses a harmless virus with some of its DNA replaced by a copy of the human SMN1 gene.
Why is naglazyme so expensive?
Developed by BioMarin Pharmaceuticals (NASDAQ:BMRN), a company specializing in rare-disease research, Naglazyme costs close to a half-million dollars annually and treats mucopolysaccharidosis type VI, which is better known as Maroteaux-Lamy syndrome, a progressive condition that causes a person’s organs to enlarge and …
What is Hutterite Syndrome?
Bowen Hutterite syndrome is primarily characterized by distinctive malformations of the head and facial (craniofacial) area as well as additional skeletal, genital, kidney (renal), and/or brain abnormalities.
What should be the infusion rate for Elaprase?
The infusion rate should not exceed 100 mL per hour. The infusion rate may be slowed, temporarily stopped, or discontinued for that visit in the event of hypersensitivity reactions [see Warnings and Precautions (5.1)]. Elaprase should not be infused with other products in the infusion tubing.
How often should I give my Child Elaprase?
The safety and efficacy of Elaprase have not been established in pediatric patients less than 16 months of age [see Use in Specific Populations (8.4) ]. The recommended dosage regimen of Elaprase is 0.5 mg per kg of body weight administered once weekly as an intravenous infusion.
How long should Elaprase be refrigerated after use?
ELAPRASE does not contain preservatives; therefore, after dilution with saline, the infusion bags should be used immediately. If immediate use is not possible, the diluted solution should be stored refrigerated at 2°C to 8°C (36°F to 46 °F) for up to 24 hours.
What are the side effects of Elaprase in humans?
Elaprase – Clinical Pharmacology 1 Mechanism of Action. Hunter syndrome (Mucopolysaccharidosis II, MPS II) is an X-linked recessive disease caused by insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. 2 Pharmacodynamics. Decreases in urinary GAG levels were observed following treatment with Elaprase. 3 Pharmacokinetics.