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 |
|---|---|---|---|---|
| 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
Created
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Wed Apr 02 06:53:38 GMT 2025
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| Nucleic Acid Type | OLIGONUCLEOTIDE |
| Record UNII |
2H1PA8H1EN
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| Record Status |
Validated (UNII)
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| Record Version |
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Preferred Name | English | ||
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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 |
| 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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admin on Wed Apr 02 06:53:38 GMT 2025 , Edited by admin on Wed Apr 02 06:53:38 GMT 2025
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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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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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4905
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SUB25196
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100000089152
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2H1PA8H1EN
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PRIMARY |
| Sugar | Site Range | Site Count |
|---|---|---|
| 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 |
|---|---|---|
| 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 |
|---|---|---|---|---|---|---|
| NUCLEOTIDE SUBSTITUTION | [1_28] | THYMIDINE 3'-MONOPHOSPHATE | Q84LAH3NDH | |||
| PEGYLATION | [1_1] | SITE_SPECIFIC | PEGAPTANIB, PEGYLATED 2'-DEOXY-2'-FLUOROURIDINE 5'-MONOPHOSPHATE- | 0E3EGS14RZ |