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Identification
- Summary
Alglucosidase alfa is an acid alpha- glucosidase (GAA) derivative used as an enzyme replacement therapy for the treatment of Pompe disease in infants and pediatric patients caused by GAA deficiency.
- Brand Names
- Lumizyme, Myozyme
- Generic Name
- Alglucosidase alfa
- DrugBank Accession Number
- DB01272
- Background
Aglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes. It is encoded by the most predominant of nine observed haplotypes of this gene. Aglucosidase alfa is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Alglucosidase alfa degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen. Structurally, Alglucosidase alfa is a glycoprotein with a calculated mass of 98,008 daltons for the 883 residue mature polypeptide chain, and a total mass of approximately 109,000 daltons, including carbohydrates. It is used for the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
- Type
- Biotech
- Groups
- Approved
- Biologic Classification
- Protein Based Therapies
Recombinant Enzymes - Protein Structure
- Protein Chemical Formula
- C4435H6739N1175O1279S32
- Protein Average Weight
- 105270.802 Da
- Sequences
>Alglucosidase alfa AHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGAQMGQPWCFF PPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLHFTIKDPANR RYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLFFADQFLQLS TSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLALEDGGSAHG VFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGYPFMPPYWGL GFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDGFRDFPAMVQ ELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKVWPGSTAFPD FTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELENPPYVPGVVG GTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISRSTFAGHGRY AGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVRWTQLGAFYP FMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAGETVARPLFL EFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPVEALGSLPPP PAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQPMALAVALTK GGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQLQKVTVLGV ATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC
Download FASTA Format- Synonyms
- Acid maltase
- Acid-alpha glucosidase
- Aglucosidase alfa
- Aglucosidase alpha
- Alglucosidasa alfa
- Alglucosidase alfa
- alpha-1,4-glucosidase
- Alpha-glucosidase
- Human acid precursor alpha-glucosidase, recombinant
- Lysosomal Alpha-Glucosidase
Pharmacology
- Indication
For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Management of Pompe's disease •••••••••••• - Contraindications & Blackbox Warnings
Prevent Adverse Drug Events TodayTap into our Clinical API for life-saving information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.Avoid life-threatening adverse drug events with our Clinical API- Pharmacodynamics
Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. In the infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac and skeletal muscles, and hepatic tissues, leading to the development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function. In the juvenile- and adult-onset forms, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness. Death in all forms is usually related to respiratory failure. Alglucosidase alfa provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen.
- Mechanism of action
Alglucosidase alfa is designed to act as an exogenous source of GAA, acting to correct GAA deficiency that is the hallmark of Pompe disease. Alglucosidase alfa binds to mannose-6-phosphate receptors on the cell surface via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen. Specifically, it hydrolyses alpha-1,4-glucose bonds.
Target Actions Organism ACation-dependent mannose-6-phosphate receptor binderHumans AGlycogen cleavageHumans - Absorption
Not Available
- Volume of distribution
- 96 ± 16 mL/kg [20 mg/kg dose]
- 119 ± 28 mL/kg [40 mg/kg dose]
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Via kidney and liver
- Half-life
2.3 ± 0.4 hours.
- Clearance
- 25+/- 4 mL/hr/kg [4-hour IV infusion of 20 mg/kg]
- Adverse Effects
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There have been no reports of overdose with alglucosidase alfa.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.No interactions found.
- Food Interactions
- No interactions found.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Lumizyme Injection, powder, for solution 5 mg/1mL Intravenous Genzyme Corporation 2010-05-24 Not applicable US 
Myozyme Injection, powder, for solution 50 mg Intravenous Sanofi S.R.L. 2016-09-20 Not applicable EU 
Myozyme Injection, powder, for solution 50 mg Intravenous Sanofi S.R.L. 2016-09-20 Not applicable EU Myozyme Powder, for solution 50 mg / vial Intravenous Sanofi Aventis Deutschland Gmb H 2006-12-06 Not applicable Canada 
Myozyme Injection, powder, for solution 50 mg Intravenous Sanofi S.R.L. 2016-09-20 Not applicable EU
Categories
- ATC Codes
- A16AB07 — Alglucosidase alfa
- Drug Categories
- Chemical TaxonomyProvided by Classyfire
- Description
- Not Available
- Kingdom
- Organic Compounds
- Super Class
- Organic Acids
- Class
- Carboxylic Acids and Derivatives
- Sub Class
- Amino Acids, Peptides, and Analogues
- Direct Parent
- Peptides
- Alternative Parents
- Not Available
- Substituents
- Not Available
- Molecular Framework
- Not Available
- External Descriptors
- Not Available
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- DTI67O9503
- CAS number
- 420784-05-0
References
- General References
- Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE: Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 2007 Jan 9;68(2):99-109. Epub 2006 Dec 6. [Article]
- External Links
- KEGG Drug
- D03207
- PubChem Substance
- 46504595
- 629565
- ChEMBL
- CHEMBL1201824
- Therapeutic Targets Database
- DAP001289
- PharmGKB
- PA164754753
- RxList
- RxList Drug Page
- Drugs.com
- Drugs.com Drug Page
- Wikipedia
- Alglucosidase_alfa
- FDA label
- Download (1.11 MB)
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample dataNot Available Approved for Marketing Not Available Acid Maltase Deficiency Disease / Glycogen Storage Disease Type II / Glycogenosis 2 / Late-onset Pompe Disease 1 somestatus stop reason just information to hide Not Available Approved for Marketing Not Available Glycogen Storage Disease Type II / Glycogenosis 2 1 somestatus stop reason just information to hide Not Available Completed Not Available Glycogen Storage Disease Type II 2 somestatus stop reason just information to hide Not Available Completed Not Available Glycogen Storage Disease Type II / Glycogen Storage Disorders 1 somestatus stop reason just information to hide Not Available Completed Not Available Type 2 Diabetes Mellitus 3 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Genzyme Inc.
- Dosage Forms
Form Route Strength Injection, powder, for solution Intravenous 5 mg/1mL Injection, powder, for solution Intravenous 50 MG Injection, powder, lyophilized, for solution Intravenous 5 mg/1mL Powder, for solution Intravenous 50 mg / vial Injection, powder, for solution Intravenous Injection, powder, lyophilized, for solution Intravenous 52.5 mg Injection, powder, lyophilized, for solution Intravenous 50 mg Powder 50 mg/1vial - Prices
Unit description Cost Unit Myozyme 50 mg vial 720.0USD vial DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.- Patents
Patent Number Pediatric Extension Approved Expires (estimated) Region CA2416492 No 2008-04-29 2021-07-10 Canada
Properties
- State
- Liquid
- Experimental Properties
- Not Available
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Yes
- Actions
- Binder
- General Function
- Transport of phosphorylated lysosomal enzymes from the Golgi complex and the cell surface to lysosomes. Lysosomal enzymes bearing phosphomannosyl residues bind specifically to mannose-6-phosphate receptors in the Golgi apparatus and the resulting receptor-ligand complex is transported to an acidic prelyosomal compartment where the low pH mediates the dissociation of the complex
- Specific Function
- protein domain specific binding
- Gene Name
- M6PR
- Uniprot ID
- P20645
- Uniprot Name
- Cation-dependent mannose-6-phosphate receptor
- Molecular Weight
- 30993.06 Da
References
- Chavez CA, Bohnsack RN, Kudo M, Gotschall RR, Canfield WM, Dahms NM: Domain 5 of the cation-independent mannose 6-phosphate receptor preferentially binds phosphodiesters (mannose 6-phosphate N-acetylglucosamine ester). Biochemistry. 2007 Nov 6;46(44):12604-17. Epub 2007 Oct 10. [Article]
References
- Hu D, Kamiya Y, Totani K, Kamiya D, Kawasaki N, Yamaguchi D, Matsuo I, Matsumoto N, Ito Y, Kato K, Yamamoto K: Sugar-binding activity of the MRH domain in the ER alpha-glucosidase II beta subunit is important for efficient glucose trimming. Glycobiology. 2009 Oct;19(10):1127-35. doi: 10.1093/glycob/cwp104. Epub 2009 Jul 22. [Article]
- Christiansen C, Hachem MA, Glaring MA, Vikso-Nielsen A, Sigurskjold BW, Svensson B, Blennow A: A CBM20 low-affinity starch-binding domain from glucan, water dikinase. FEBS Lett. 2009 Apr 2;583(7):1159-63. doi: 10.1016/j.febslet.2009.02.045. Epub 2009 Mar 9. [Article]
Drug created at May 16, 2007 20:19 / Updated at June 26, 2024 02:22