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Macugen (Pegaptanib Sodium)
Macugen (Pegaptanib Sodium) is a VEGF inhibitor / Aptamer Small molecule drug developed by Retina Institute of Hawaii. It is currently FDA-approved for Neovascular (wet) age-related macular degeneration. Also known as: Macugen® (pegaptanib sodium).
Pegaptanib is an aptamer that binds and inhibits vascular endothelial growth factor (VEGF), reducing abnormal blood vessel growth in the eye.
Pegaptanib is an aptamer that binds and inhibits vascular endothelial growth factor (VEGF), reducing abnormal blood vessel growth in the eye. Used for Neovascular (wet) age-related macular degeneration.
At a glance
| Generic name | Macugen (Pegaptanib Sodium) |
|---|---|
| Also known as | Macugen® (pegaptanib sodium) |
| Sponsor | Retina Institute of Hawaii |
| Drug class | VEGF inhibitor / Aptamer |
| Target | VEGF165 (Vascular Endothelial Growth Factor) |
| Modality | Small molecule |
| Therapeutic area | Ophthalmology |
| Phase | FDA-approved |
Mechanism of action
Pegaptanib is a pegylated aptamer—a short, single-stranded DNA molecule—that selectively binds to VEGF165, a key isoform of vascular endothelial growth factor. By neutralizing VEGF, it suppresses neovascularization and vascular permeability in the retina, slowing vision loss in age-related macular degeneration. It is administered as an intravitreal injection.
Approved indications
- Neovascular (wet) age-related macular degeneration
Common side effects
- Conjunctival hemorrhage
- Eye pain
- Floaters
- Photopsia (flashing lights)
- Anterior chamber inflammation
Key clinical trials
- Pegaptanib Therapy in Non-Infectious Uveitic Cystoid Macular Edema (NA)
- Extension Study to Gather Data on Effect of Macugen on the Corneal Endothelium (PHASE4)
- A Study On The Efficacy Of Macugen Injections In Patients With Neovascular Age-Related Macular Degeneration In Real Life.
- A Multi-Center Trial To Evaluate The Safety And Efficacy Of Pegaptanib Sodium(Macugen) Injected Into The Eye Every 6 Weeks For Up To 2 Years For Macular Swelling Associated With Diabetes, With An Open-Label Macugen Year Extension. (PHASE2, PHASE3)
- Cohort Study of the Clinical Course of Macular Diseases in Kagawa (PHASE4)
- Cohort Study of the Clinical Course of Macular Diseases in Japanese
- A Safety and Efficacy Study Comparing the Combination Treatments of Verteporfin Therapy Plus One of Two Different Doses of Intravitreal Triamcinolone Acetonide and the Verteporfin Therapy Plus Intravitreal Pegaptanib (PHASE3)
- Long-Term Use Of Pegaptanib In Patients For Subfoveal Choroidal Neovascularization Secondary To Age-Related Macular Degeneration
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Macugen (Pegaptanib Sodium) CI brief — competitive landscape report
- Macugen (Pegaptanib Sodium) updates RSS · CI watch RSS
- Retina Institute of Hawaii portfolio CI
Frequently asked questions about Macugen (Pegaptanib Sodium)
What is Macugen (Pegaptanib Sodium)?
How does Macugen (Pegaptanib Sodium) work?
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Who makes Macugen (Pegaptanib Sodium)?
Is Macugen (Pegaptanib Sodium) also known as anything else?
What drug class is Macugen (Pegaptanib Sodium) in?
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What does Macugen (Pegaptanib Sodium) target?
Related
- Drug class: All VEGF inhibitor / Aptamer drugs
- Target: All drugs targeting VEGF165 (Vascular Endothelial Growth Factor)
- Manufacturer: Retina Institute of Hawaii — full pipeline
- Therapeutic area: All drugs in Ophthalmology
- Indication: Drugs for Neovascular (wet) age-related macular degeneration
- Also known as: Macugen® (pegaptanib sodium)
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing