アルグルコシダーゼアルファ
- 関
- human acid alpha-glucosidase、recombinant human acid alpha-glucosidase、rhGAA
Wikipedia preview
出典(authority):フリー百科事典『ウィキペディア（Wikipedia）』「2014/12/21 03:32:55」(JST)
[Wiki en表示]
Alglucosidase alfa
| Systematic (IUPAC) name |
| Human glucosidase, prepro-α-[199-arginine,223-histidine] [1] |
| Clinical data |
| AHFS/Drugs.com |
monograph |
| Legal status |
- FDA approved for children[2]
|
| Routes |
Intravenous[2] |
| Identifiers |
| CAS number |
420794-05-0 N |
| ATC code |
A16AB07 |
| DrugBank |
DB01272 |
| UNII |
DTI67O9503 N |
| KEGG |
D03207 N |
| Chemical data |
| Formula |
C4758H7262N1274O1369S35 [1] |
| Mol. mass |
105338 [1] |
| N (what is this?) (verify) |
Alglucosidase alfa (Lumizyme, Myozyme, Genzyme) is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD).[3] Chemically speaking, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease.[2]
Contents
- 1 Status
- 2 Side effects
- 3 References
- 4 External links
Status
Orphan drug pharmaceutical company, Genzyme, markets alglucosidase alfa as "Myozyme". In 2006, the U.S. Food and Drug Administration (FDA) approved Myozyme as a suitable ERT treatment for children.[2] Some health plans have refused to subsidize Myozyme for adult patients because it lacks approval for treatment in adults, as well as its high cost (US$300,000/yr for life).[4]
On August 1, 2014 the U.S. Food and Drug Administration announced the approval of Lumizyme (alglucosidase alfa) for treatment of patients with infantile-onset Pompe disease, including patients who are less than 8 years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) known as the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program is being eliminated. [5]
Side effects
Common observed adverse reactions to alglucosidase alfa treatment are pneumonia, respiratory complications, infections and fever. More serious reactions reported include heart and lung failure and allergic shock. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.[2]
References
- ^ a b c American Medical Association (USAN). "Alglucosidase alfa" (Microsoft Word). STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL. Retrieved 18 December 2007.
- ^ a b c d e "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. Retrieved 2008-07-07.
- ^ Kishnani PS, Corzo D, Nicolino M et al. (2007). "Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease". Neurology 68 (2): 99–109. doi:10.1212/01.wnl.0000251268.41188.04. PMID 17151339.
- ^ Geeta Anand (2007-09-18). "As Costs Rise, New Medicines Face Pushback". Wall Street Journal (Dow Jones & Company). Retrieved 2008-07-07.
- ^ cite press release |title=FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements |publisher=FDA |date=2014-08-14 |url=http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407563.htm
External links
- Myozyme (alglucosidase alfa), Genzyme official website
- MTAP (Myozyme Temporary Access Program), Genzyme official website
|
Other alimentary tract and metabolism products (A16)
|
|
| Amino acids and derivatives |
- Levocarnitine
- Ademetionine
- Levoglutamide
- Cysteamine
- Carglumic acid
- Betaine
|
|
| Enzymes |
- Carbohydrate metabolism: sucrase (Sacrosidase)
- alpha-glucosidase (Alglucosidase alfa)
- Glycolipid/sphingolipid: glucocerebrosidase (Alglucerase
- Imiglucerase
- Taliglucerase alfa
- Velaglucerase alfa)
- alpha-galactosidase (Agalsidase alfa
- Agalsidase beta)
- Glycosaminoglycan: iduronidase (Laronidase)
- arylsulfatase B (Galsulfase)
- iduronate-2-sulfatase (Idursulfase)
|
|
Various alimentary tract
and metabolism products |
- Anethole trithione
- Eliglustat
- Glycerol phenylbutyrate
- Miglustat
- Nitisinone
- Sapropterin
- Sodium phenylbutyrate
- Teduglutide
- Tioctic acid
- Zinc acetate
|
|
|
|
anat (t, g, p)/phys/devp/enzy
|
noco/cong/tumr, sysi/epon
|
proc, drug (A2A/2B/3/4/5/6/7/14/16), blte
|
|
|
|
UpToDate Contents
全文を閲覧するには購読必要です。 To read the full text you will need to subscribe.
English Journal
- Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease.
- Rafael Bretón Martínez J1, Martínez AC2.
- Journal of the neurological sciences.J Neurol Sci.2015 Nov 15;358(1-2):459-60. doi: 10.1016/j.jns.2015.08.007. Epub 2015 Aug 7.
- PMID 26279333
- A study of up to 12 years of follow-up of Friedreich ataxia utilising four measurement tools.
- Tai G1, Corben LA2, Gurrin L3, Yiu EM4, Churchyard A5, Fahey M6, Hoare B5, Downie S5, Delatycki MB7.
- Journal of neurology, neurosurgery, and psychiatry.J Neurol Neurosurg Psychiatry.2015 Jun;86(6):660-6. doi: 10.1136/jnnp-2014-308022. Epub 2014 Aug 11.
- OBJECTIVE: To explore the progression of Friedreich ataxia by analysing the change in scores of four clinical measures (the Friedreich Ataxia Rating Scale (FARS), the International Cooperative Ataxia Rating Scale (ICARS), the Functional Independence Measure (FIM) and the Modified Barthel Index (MBI)
- PMID 25112308
- Novel GAA mutations in patients with Pompe disease.
- Turaça LT1, de Faria DO1, Kyosen SO2, Teixeira VD1, Motta FL1, Pessoa JG1, Rodrigues E Silva M1, de Almeida SS1, D'Almeida V3, Munoz Rojas MV4, Martins AM2, Pesquero JB5.
- Gene.Gene.2015 Apr 25;561(1):124-31. doi: 10.1016/j.gene.2015.02.023. Epub 2015 Feb 12.
- Pompe disease is an autosomal recessive disorder linked to GAA gene that leads to a multi-system intralysosomal accumulation of glycogen. Mutation identification in the GAA gene can be very important for early diagnosis, correlation between genotype-phenotype and therapeutic intervention. For this p
- PMID 25681614
Japanese Journal
- Binding with parameters and thermodynamics of the interaction of imino sugars a recombinant human acid alpha-glucosidase (alglucosidase alfa): Insight into the complex formation mechanism
Related Links
- Alglucosidase Alfa reference guide for safe and effective use from the American Society of Health-System Pharmacists (AHFS DI). ... In the infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in ...
- Posts about Alglucosidase alfa written by mtariqniaz ... MEDICATION SAFETY ISSUES Sound-alike/look-alike issues: Alglucosidase alfa may be confused with agalsidase alfa, agalsidase beta, alglucerase SPECIAL ALERTS
★リンクテーブル★
[★]
- 英
- alglucosidase alfa
- 商
- マイオザイム
- 関
- ヒト酸性αグルコシダーゼ、組換え型ヒト酸性αグルコシダーゼ。糖原病
II型糖原病
[★]
- 関
- alglucosidase alfa、human acid alpha-glucosidase、recombinant human acid alpha-glucosidase
[★]
ヒト酸性αグルコシダーゼ
- 関
- alglucosidase alfa、recombinant human acid alpha-glucosidase、rhGAA
[★]
組換え型ヒト酸性αグルコシダーゼ
- 関
- alglucosidase alfa、human acid alpha-glucosidase、rhGAA
[★]
- 関
- alpha