Idursulfase
| Clinical data | |
|---|---|
| Drug class | Enzyme[1] |
| Main uses | Mucopolysaccharidosis II (MPS-II)[1] |
| Side effects | Rash, fever, headache, high blood pressure, flushing[2] |
| Pregnancy category |
|
| Typical dose | 0.5 mg/kg q 1 week[3] |
| External links | |
| AHFS/Drugs.com | Monograph |
| Legal | |
| License data | |
Idursulfase, sold under the brand name Elaprase, is a medication used to treat mucopolysaccharidosis II (MPS-II, Hunter syndrome).[1] It is given by gradual injection into a vein.[1] It is used long term.[3] Effects on life expectancy and quality of life is unclear.[4]
Common side effects include rash, fever, headache, high blood pressure, and flushing.[2] Other side effects may include shortness of breath, heartburn, chest pain, and anaphylaxis.[2] It is a form of lysosomal enzyme known as iduronate-2-sulfatase and is made by recombinant DNA technology.[1]
Idursulfase was approved for medical use in the United States in 2006 and Europe in 2007.[1][2] In the United States it costs about 475,000 USD to treat a 35 kg person for a year.[5] In the United Kingdom this amount costs £310,000.[3]
Medical uses
Dosage
It is given at a dose of 0.5 mg/kg once per week.[3]
Mechanism of action
In terms of the mode of action of idursulfase we find that it involves the hydrolysis of 2-sulfate esters of terminal iduronate sulfate residues from GAGs. This in turn reduces the accumulation of GAGs(which otherwise causes cellular engorgement, organomegaly, and organ system dysfunction)[6][7]
History
Timeline of important aspects in clinical development of idursulfase beginning in the year 2001 until recently[6](regulatory milestones and initiation dates of ongoing clinical studies are indicated with white boxes with red border)
References
- ↑ 1.0 1.1 1.2 1.3 1.4 1.5 1.6 "Idursulfase Monograph for Professionals". Drugs.com. Archived from the original on 2 December 2021. Retrieved 25 November 2021.
- ↑ 2.0 2.1 2.2 2.3 "Elaprase". Archived from the original on 11 December 2021. Retrieved 25 November 2021.
- ↑ 3.0 3.1 3.2 3.3 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1116. ISBN 978-0857114105.
- ↑ "Idursulfase" (PDF). September 2009. Archived (PDF) from the original on 15 April 2021. Retrieved 25 November 2021.
- ↑ "Idursulfase Prices, Coupons & Savings Tips - GoodRx". GoodRx. Retrieved 25 November 2021.
- ↑ 6.0 6.1 6.2 Whiteman, David AH; Kimura, Alan (23 August 2017). "Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future". Drug Design, Development and Therapy. 11: 2467–2480. doi:10.2147/DDDT.S139601.
- ↑ "ELAPRASE (idursulfase)" (PDF). fda. Archived (PDF) from the original on 29 September 2024. Retrieved 2 February 2025.
External links
| Identifiers: |
|
|---|
- idursulfase at the US National Library of Medicine Medical Subject Headings (MeSH)
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