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出典(authority):フリー百科事典『ウィキペディア（Wikipedia）』「2014/03/29 11:07:16」(JST)

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Imiglucerase is a medication used in the treatment of Gaucher's disease.^[1]^[2]

It is a recombinant DNA-produced analogue of human β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation. It is given intravenously after reconstitution as a treatment for Type 1 Gaucher's disease. It is available in formulations containing 200 or 400 units per vial. The specific activity of highly purified human enzyme is 890,000 units/mg.^[3] A typical dose is 2.5U/kg every two weeks, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up.^[4] It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000.^[5] Due to the low profitability (and high expense) of developing medications for rare conditions, imiglucerase has been granted orphan drug status in the USA, Australia, and Japan.^[6]

Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2117.^[7]

References[edit]

^ Weinreb NJ (August 2008). "Imiglucerase and its use for the treatment of Gaucher's disease". Expert Opin Pharmacother 9 (11): 1987–2000. doi:10.1517/14656566.9.11.1987. PMID 18627336.
^ Starzyk K, Richards S, Yee J, Smith SE, Kingma W (February 2007). "The long-term international safety experience of imiglucerase therapy for Gaucher disease". Mol. Genet. Metab. 90 (2): 157–63. doi:10.1016/j.ymgme.2006.09.003. PMID 17079176.
^ Pentchev et al.; Brady, RO; Blair, HE; Britton, DE; Sorrell, SH (August 1978). "Gaucher disease: Isolation and comparison of normal and mutant glucocerebrosidase from human spleen tissue". Proc. Natl. Acad. Sci. USA 75 (8): 3970–3973. doi:10.1073/pnas.75.8.3970. PMC 392911. PMID 29293.
^ "Cerezyme (imiglucerase for injection) Genzyme product data sheet".
^ Balancing innovation, access, and profits -- marketing exclusivity for biologics, Alfred B. Engelberg et al., N Engl J Med 361:1917
^ "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs.".
^ Erin Ailworth and Robert Weisman (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe.

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1. ゴーシェ病：治療 gaucher disease treatment
2. 薬物療法の最新情報 whats new in drug therapy
3. 血液学の最新情報 whats new in hematology

English Journal

Glucocerebrosidase Enhancers for Selected Gaucher Disease Genotypes by Modification of α-1-C-Substituted Imino-D-xylitols (DIXs) by Click Chemistry.

Serra-Vinardell J1, Díaz L, Casas J, Grinberg D, Vilageliu L, Michelakakis H, Mavridou I, Aerts JM, Decroocq C, Compain P, Delgado A.
ChemMedChem.ChemMedChem.2014 Aug;9(8):1744-54. doi: 10.1002/cmdc.201402023. Epub 2014 Jun 27.
A series of hybrid analogues was designed by combination of the iminoxylitol scaffold of parent 1C9-DIX with triazolylalkyl side chains. The resulting compounds were considered potential pharmacological chaperones in Gaucher disease. The DIX analogues reported here were synthesized by CuAAC click ch
PMID 24976039

Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model.

Vigan M1, Stirnemann J, Caillaud C, Froissart R, Boutten A, Fantin B, Belmatoug N, Mentré F.
Orphanet journal of rare diseases.Orphanet J Rare Dis.2014 Jun 30;9:95. doi: 10.1186/1750-1172-9-95.
BACKGROUND: Gaucher disease (GD) is a rare recessively inherited disorder caused by deficiency of a lysosomal enzyme, glucocerebrosidase. Accumulation of glucosylceramide or glucosylsphingosine in macrophages leads to increased production of ferritin and chitotriosidase and to decreases in hemoglobi
PMID 24980507

A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.

Pastores GM1, Petakov M2, Giraldo P3, Rosenbaum H4, Szer J5, Deegan PB6, Amato DJ7, Mengel E8, Tan ES9, Chertkoff R10, Brill-Almon E11, Zimran A12.
Blood cells, molecules & diseases.Blood Cells Mol Dis.2014 Jun 17. pii: S1079-9796(14)00055-2. doi: 10.1016/j.bcmd.2014.05.004. [Epub ahead of print]
Taliglucerase alfa is a β-glucosidase enzyme replacement therapy (ERT) approved in the US and other countries for the treatment of Gaucher disease (GD) in adults and is approved in pediatric and adult patients in Australia and Canada. It is the first approved plant cell-expressed recombinant human
PMID 24950666

Japanese Journal

Successful Pregnancy and Lactation Outcome in a Patient With Gaucher Disease Receiving Enzyme Replacement Therapy, and the Subsequent Distribution and Excretion of Imiglucerase in Human Breast Milk

Sekijima Yoshiki,Ohashi Toya,Kosho Tomoki,Fukushima Yoshimitsu
CLINICAL THERAPEUTICS 32(12), 2048-2052, 2010-11
… Background: Enzyme replacement therapy (ERT) with imiglucerase is a well-established, effective treatment for Gaucher disease. … However, there have been no published reports regarding the excretion of imiglucerase into human breast milk and its effects on the nursing infant. …
NAID 120005248172

Gaucher 病酵素補充療法の変更後, 多発骨壊死をきたした1例

阿部真美,蓑口まどか,神保絢子 [他],菊地陽子,千葉篤,柴田好,高後裕
日本内科学会雑誌 93(11), 2421-2423, 2004-11-10
NAID 10014293632

Improvement of Splenomegaly and Pancytopenia by Enzyme Replacement Therapy Against Type 1 Gaucher Disease: A Report of Sibling Cases

TSUBOI Kazuya,IIDA Shinsuke,KATO Miyuki,HAYAMI Yoshihito,HANAMURA Ichiro,MIURA Kazuhisa,HARADA Shinsuke,KOMATSU Hirokazu,BANNO Shogo,WAKITA Atsushi,NITTA Masakazu,UEDA Ryuzo
International journal of hematology 73(3), 356-362, 2001-04
NAID 10007320711

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リンク元	「イミグルセラーゼ」

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Imiglucerase
Systematic (IUPAC) name
	Human Beta-glucocerebrosidase
Clinical data
AHFS/Drugs.com	monograph
MedlinePlus	a601149
Licence data	EMA:Link, US FDA:link
Legal status	?
Routes	Intravenous
Pharmacokinetic data
Half-life	3.6-10.4 min
Identifiers
CAS number	143003-46-7
ATC code	A16AB02
DrugBank	DB00053
UNII	Q6U6J48BWY
KEGG	D02810
ChEMBL	CHEMBL1201632
Chemical data
Formula	C2532H3854N672O711S16
Mol. mass	55597.4 g/mol (unglycosylated)
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英: imiglucerase
商: セレザイム
関: ゴーシェ病

マクロファージに蓄積する糖脂質グルコセレブロシド分解酵素の改良型でゴーシェ病に適応

[pmid18627336-1] Weinreb NJ (August 2008). "Imiglucerase and its use for the treatment of Gaucher's disease". Expert Opin Pharmacother 9 (11): 1987–2000. doi:10.1517/14656566.9.11.1987. PMID 18627336.

[pmid17079176-2] Starzyk K, Richards S, Yee J, Smith SE, Kingma W (February 2007). "The long-term international safety experience of imiglucerase therapy for Gaucher disease". Mol. Genet. Metab. 90 (2): 157–63. doi:10.1016/j.ymgme.2006.09.003. PMID 17079176.

[3] Pentchev et al.; Brady, RO; Blair, HE; Britton, DE; Sorrell, SH (August 1978). "Gaucher disease: Isolation and comparison of normal and mutant glucocerebrosidase from human spleen tissue". Proc. Natl. Acad. Sci. USA 75 (8): 3970–3973. doi:10.1073/pnas.75.8.3970. PMC 392911. PMID 29293.

[4] "Cerezyme (imiglucerase for injection) Genzyme product data sheet".

[5] Balancing innovation, access, and profits -- marketing exclusivity for biologics, Alfred B. Engelberg et al., N Engl J Med 361:1917

[6] "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs.".

[7] Erin Ailworth and Robert Weisman (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe.

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imiglucerase