Pegaptanib is a selective vascular endothelial growth factor (VEGF) antagonist indicated for the treatment of neovascular (wet) age-related macular degeneration. Pegaptanib is an aptamer, a pegylated modified single-stranded oligonucleotide, which adopts a threedimensional conformation that enables it to bind to extracellular VEGF. Pegaptanib specifically binds to the 165 isoform of VEGF, a protein that plays a critical role in angiogenesis (the formation of new blood vessels) and increased permeability (leakage from blood vessels), two of the primary pathological processes responsible for the vision loss associated with neovascular age-related macular degeneration. Pegaptanib is administered in a 0.3 mg dose once every six weeks by intravitreal injection. An intravitreal injection is one that is administered directly into the eye, more specifically, into the vitreous humour, or the jelly-like fluid within the eye.
Approval Year
Conditions
Condition | Modality | Targets | Highest Phase | Product |
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Primary | MACUGEN Approved UseMacugen is indicated for the treatment of neovascular (wet) age-related macular degeneration. Macugen is indicated for the treatment of neovascular (wet) age-related macular degeneration (1). Launch Date2004 |
Substance Class |
Nucleic Acid
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Nucleic Acid Type | OLIGONUCLEOTIDE |
Nucleic Acid Subtype | |
Record UNII |
2H1PA8H1EN
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Record Status |
Validated (UNII)
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Record Version |
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Official Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Common Name | English |
Classification Tree | Code System | Code | ||
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WHO-VATC |
QS01LA03
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NCI_THESAURUS |
C1742
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NDF-RT |
N0000178292
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WHO-ATC |
S01LA03
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EMA ASSESSMENT REPORTS |
MACUGEN (AUTHORIZED: WET MACULAR DEGENERATION)
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Code System | Code | Type | Description | ||
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C90657
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PRIMARY | |||
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N0000170810
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PRIMARY | Aptamers, Nucleotide [Chemical/Ingredient] | ||
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8215
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PRIMARY | |||
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498509
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PRIMARY | RxNorm | ||
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N0000011393
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PRIMARY | Oligonucleotides [Chemical/Ingredient] | ||
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CHEMBL2108752
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PRIMARY | |||
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6836
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PRIMARY | |||
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DB04895
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PRIMARY | |||
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C495058
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PRIMARY | |||
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PEGAPTANIB
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PRIMARY | |||
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4905
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PRIMARY | |||
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SUB25196
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PRIMARY | |||
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2H1PA8H1EN
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PRIMARY |
Sugar | Site Range | Site Count |
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D-Ribose (RNA) | 1_4-1_5 | 2 / 28 |
Deoxyribose (DNA) | 1_28 | 1 / 28 |
2'-O-Methyl-Ribose | 1_2-1_3;1_8-1_9;1_11-1_13;1_15;1_19;1_21;1_23;1_27 | 12 / 28 |
2'-Fluoro-Ribose | 1_1;1_6-1_7;1_10;1_14;1_16-1_18;1_20;1_22;1_24-1_26 | 13 / 28 |
Linkage | Site Range | Site Count |
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Phosphate | 1_1-1_27 | 27 / 27 |
Related Record | Type | Details | ||
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TARGET -> INHIBITOR |
Related Record | Type | Details | ||
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ACTIVE MOIETY |
Structural Modifications
Modification Type | Location Site | Location Type | Residue Modified | Extent | Fragment Name | Fragment Approval |
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NUCLEOTIDE SUBSTITUTION | [1_28] | THYMIDINE 3'-MONOPHOSPHATE | Q84LAH3NDH | |||
PEGYLATION | [1_1] | SITE_SPECIFIC | PEGAPTANIB, PEGYLATED 2'-DEOXY-2'-FLUOROURIDINE 5'-MONOPHOSPHATE- | 0E3EGS14RZ |