TABLE 57.1.

Examples of glycan-based drugs, their target diseases, and modes of action

DrugSourceTarget diseaseMode of action
Targeting sialic acids
 zanamivir (Relenza)Biota/GlaxoSmithKlineinfluenza type A and Binhibits neuraminidase
 oseltamivir (GS 4104, Tamiflu)Gilead/Rochechemoprophylaxisinhibits neuraminidase
Targeting glycosaminoglycans
 heparinmultiple brandsanticoagulant; possible value in cancer metastasis preventionactivates antithrombin; inhibits heparanase and selectins and blocks interactions between growth factors and heparan sulfate
 hyaluronan (HA)multiple brandsocular surgery; osteoarthritis; plastic surgerytissue space filler; anti-inflammatory agent
 laronidase (Aldurazyme)Genzymemucopolysaccharidosis type I (MPSI); α-idu-ronidase deficiencyERT
 galsulfase (Naglazyme)Biomarinmucopolysaccharidosis type VI; arylsulfatase B deficiencyERT
 hyaluronidase (Cumulase)Halozymein vitro fertilization; in development as an adjuvant for cancer chemotherapydegrades HA around oocytes improving fertilization; degrades HA in tumors to decrease intratumor pressure
 ALZ-801phase III trials (Alzheon, Inc.)amyloid diseases, Alzheimer's disease, and possibly other amyloidosesbinds to amyloid plaque, blocks its formation
Targeting glycosphingolipids
N-butyl-deoxynojirimycin (DNJ) (Miglustat, Zavesca) Aceteliontype 1 Gaucher's disease; Niemann–Pick's disease type C; late-onset Tay–Sach's disease; type 3 Gaucher's diseasesubstrate reduction therapy; inhibits glucosylceramide synthase
 imiglucerase (Cerezyme)Genzymetype 1 Gaucher's diseaseERT
 β-agalsidase (Fabrazyme)GenzymeFabry disease; α-galactosidase A deficiencyERT
Others
 acarbose (Glucobay)Bayertype 2 diabetesblocks intestinal α-glucosidases involved in digestion of dietary glycans
 alglucosidase alfa (Myozyme)GenzymePompe's disease (glycogen storage disease); α-glucosidase A deficiencyERT
 allosamidinIndustrial Researchinsecticidechitinase inhibitor

Modified and updated from Brown JR, Crawford BE, Esko JD. 2007. Crit Rev Biochem Mol Biol 24: 481–515.

Compounds targeted at microbial glycans, such as the aminoglycoside antibiotics or other inhibitors of cell wall assembly, have not been included.

(ERT) Enzyme replacement therapy.

From: Chapter 57, Glycans in Biotechnology and the Pharmaceutical Industry

Cover of Essentials of Glycobiology
Essentials of Glycobiology [Internet]. 4th edition.
Varki A, Cummings RD, Esko JD, et al., editors.
Cold Spring Harbor (NY): Cold Spring Harbor Laboratory Press; 2022.
Copyright © 2022 The Consortium of Glycobiology Editors, La Jolla, California; published by Cold Spring Harbor Laboratory Press; doi:10.1101/glycobiology.4e.57. All rights reserved.

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