{"id":"eye001","rwe":[{"pmid":"34813254","year":"2006","title":"Pegaptanib.","finding":"","journal":"","studyType":"Clinical Study"},{"pmid":"12714656","year":"2003","title":"VEGF164 is proinflammatory in the diabetic retina.","finding":"","journal":"Investigative ophthalmology & visual science","studyType":"Clinical Study"},{"pmid":"12573989","year":"2003","title":"Intrauterine hypertension decreases lung VEGF expression and VEGF inhibition causes pulmonary hypertension in the ovine fetus.","finding":"","journal":"American journal of physiology. Lung cellular and molecular physiology","studyType":"Clinical Study"},{"pmid":"12506087","year":"2003","title":"Controlled delivery of the anti-VEGF aptamer EYE001 with poly(lactic-co-glycolic)acid microspheres.","finding":"","journal":"Investigative ophthalmology & visual science","studyType":"Clinical Study"},{"pmid":"11927845","year":"2002","title":"Preclinical and phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration.","finding":"","journal":"Retina (Philadelphia, Pa.)","studyType":"Clinical Study"}],"tags":[{"label":"Small Molecule","category":"modality"},{"label":"Vascular endothelial growth factor A","category":"target"},{"label":"VEGFA","category":"gene"},{"label":"S01LA03","category":"atc"},{"label":"Active","category":"status"},{"label":"Exudative age-related macular degeneration","category":"indication"},{"label":"Valeant Pharms Llc","category":"company"},{"label":"Approved 2000s","category":"decade"}],"phase":"marketed","safety":{},"trials":[],"aliases":[],"company":"Valeant Pharms Llc","patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=EYE001","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T03:31:48.225805+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T03:31:54.551622+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=EYE001","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T03:31:55.415492+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: Vascular endothelial growth factor A antagonist","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:31:56.457413+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL1201421/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T03:31:56.116904+00:00"}},"allNames":"macugen","offLabel":[],"synonyms":["pegaptanib","pegaptanib sodium","macugen","pegaptanib octasodium","EYE001","NX1838"],"timeline":[{"date":"2004-12-17","type":"positive","source":"DrugCentral","milestone":"FDA approval (Valeant Pharms Llc)"}],"aiSummary":"Macugen (EYE001) is a small molecule drug developed by VALEANT PHARMS LLC, targeting vascular endothelial growth factor A (VEGF-A). It was approved by the FDA in 2004 for the treatment of exudative age-related macular degeneration. Macugen works by inhibiting the action of VEGF-A, a protein that promotes the growth of new blood vessels in the eye, which can lead to vision loss. The commercial status of Macugen is patented, and it is currently owned by VALEANT PHARMS LLC. Key safety considerations include potential eye inflammation and increased risk of endophthalmitis.","brandName":"Macugen","ecosystem":[{"indication":"Exudative age-related macular degeneration","otherDrugs":[{"name":"aflibercept","slug":"aflibercept","company":"Regeneron Pharmaceuticals"},{"name":"brolucizumab","slug":"brolucizumab","company":"Novartis Pharms Corp"},{"name":"faricimab","slug":"faricimab","company":"Genentech Inc"},{"name":"ranibizumab","slug":"ranibizumab","company":"Genentech"}],"globalPrevalence":900000000}],"mechanism":{"target":"Vascular endothelial growth factor A","novelty":"Follow-on","targets":[{"gene":"VEGFA","source":"DrugCentral","target":"Vascular endothelial growth factor A","protein":"Vascular endothelial growth factor A"}],"modality":"Small Molecule","explanation":"","oneSentence":"","technicalDetail":"Macugen is a small molecule inhibitor of VEGF-A, specifically binding to the extracellular domain of the VEGF receptor 1 and 2, thereby preventing the activation of downstream signaling pathways that promote angiogenesis and vascular permeability."},"commercial":{"launchDate":"2004","_launchSource":"DrugCentral (FDA 2004-12-17, VALEANT PHARMS LLC)"},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/4905","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=EYE001","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=EYE001","fields":["publications"],"source":"PubMed/NCBI"}],"_enrichedAt":"2026-03-30T02:40:39.623547","_validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T03:31:57.682867+00:00","fieldsConflicting":1,"overallConfidence":0.8},"biosimilars":[],"competitors":[{"drugName":"verteporfin","drugSlug":"verteporfin","fdaApproval":"2000-04-12","patentStatus":"Unknown","relationship":"same-class"},{"drugName":"ranibizumab","drugSlug":"ranibizumab","fdaApproval":"2006-06-30","relationship":"same-class"},{"drugName":"aflibercept","drugSlug":"aflibercept","fdaApproval":"2011-11-18","relationship":"same-class"},{"drugName":"brolucizumab","drugSlug":"brolucizumab","fdaApproval":"2019-10-07","relationship":"same-class"},{"drugName":"bevacizumab","drugSlug":"bevacizumab","fdaApproval":"2004-02-26","relationship":"same-class"},{"drugName":"faricimab","drugSlug":"faricimab","fdaApproval":"2022-01-28","relationship":"same-class"}],"genericName":"eye001","indications":{"approved":[{"name":"Exudative age-related macular degeneration","source":"DrugCentral","snomedId":414173003,"regulator":"FDA","globalPrevalence":900000000,"prevalenceMethod":"ai-extracted","prevalenceSource":"Retina, 2018 (PMID:29059101)"}],"offLabel":[],"pipeline":[]},"drugCategory":"active","labelChanges":[],"relatedDrugs":[{"drugId":"verteporfin","brandName":"verteporfin","genericName":"verteporfin","approvalYear":"2000","relationship":"same-class"},{"drugId":"ranibizumab","brandName":"ranibizumab","genericName":"ranibizumab","approvalYear":"2006","relationship":"same-class"},{"drugId":"aflibercept","brandName":"aflibercept","genericName":"aflibercept","approvalYear":"2011","relationship":"same-class"},{"drugId":"brolucizumab","brandName":"brolucizumab","genericName":"brolucizumab","approvalYear":"2019","relationship":"same-class"},{"drugId":"bevacizumab","brandName":"bevacizumab","genericName":"bevacizumab","approvalYear":"2004","relationship":"same-class"},{"drugId":"faricimab","brandName":"faricimab","genericName":"faricimab","approvalYear":"2022","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT06717035","phase":"NA","title":"Evaluation of Performance of MiYOSMART Photochromic and Clear Spectacle Lenses in Myopic Children","status":"NOT_YET_RECRUITING","sponsor":"Singapore National Eye Centre","startDate":"2025-01-02","conditions":["Myopia","Myopia Progression"],"enrollment":202,"completionDate":"2027-01-02"},{"nctId":"NCT00239928","phase":"PHASE2","title":"Clinical Study Of Pegaptanib Sodium (EYE001) For Wet-Type Age-Related Macular Degeneration","status":"COMPLETED","sponsor":"Pfizer","startDate":"2005-09","conditions":["Macular Degeneration"],"enrollment":61,"completionDate":"2008-11"},{"nctId":"NCT00056199","phase":"PHASE1","title":"EYE001 to Treat Retinal Tumors in Patients With Von Hippel-Lindau Syndrome","status":"COMPLETED","sponsor":"National Eye Institute (NEI)","startDate":"2003-03","conditions":["Hippel-Lindau Disease"],"enrollment":5,"completionDate":"2005-11"},{"nctId":"NCT00150202","phase":"PHASE3","title":"Clinical Study Of EYE001 For Wet-Type AMD (Age-Related Macular Degeneration)","status":"COMPLETED","sponsor":"Pfizer","startDate":"2004-07","conditions":["Macular Degeneration"],"enrollment":90,"completionDate":"2006-10"},{"nctId":"NCT00321997","phase":"PHASE2,PHASE3","title":"A Clinical Trial to Explore Safety and Efficacy of Different Doses of Pegaptanib Sodium, Compared to Sham, in Patients With Wet AMD.","status":"COMPLETED","sponsor":"Eyetech Pharmaceuticals","startDate":"2001-05","conditions":["Age-Related Macular Degeneration"],"enrollment":540,"completionDate":"2007-10"},{"nctId":"NCT00040313","phase":"PHASE2","title":"Pegaptanib Sodium Compared to Sham Injection in Patients With DME Involving the Center of the Macula","status":"COMPLETED","sponsor":"Eyetech Pharmaceuticals","startDate":"2002-10","conditions":["Diabetic Macular Edema"],"enrollment":0,"completionDate":"2005-02"},{"nctId":"NCT00021736","phase":"PHASE2,PHASE3","title":"Phase II/III Study of Anti-VEGF in Neovascular AMD","status":"COMPLETED","sponsor":"Eyetech Pharmaceuticals","startDate":"2001-07","conditions":["Macular Degeneration","Choroidal Neovascularization"],"enrollment":540,"completionDate":"2002-07"}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"crossReferences":{"NUI":"N0000171612","MMSL":"19106","NDDF":"008794","UNII":"2H1PA8H1EN","VUID":"4024940","VANDF":"4024940","INN_ID":"8215","RXNORM":"498509","UMLSCUI":"C1531015","chemblId":"CHEMBL1201421","ChEMBL_ID":"CHEMBL2108752","KEGG_DRUG":"D05386","DRUGBANK_ID":"DB04895","PUBCHEM_CID":"56603655","SNOMEDCT_US":"416058004","IUPHAR_LIGAND_ID":"6836","MESH_SUPPLEMENTAL_RECORD_UI":"C495058"},"formularyStatus":[],"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[{"period":"2004-","companyName":"Valeant Pharms Llc","relationship":"Original Developer"}],"publicationCount":5,"therapeuticAreas":["Ophthalmology"],"atcClassification":{"source":"DrugCentral","atcCode":"S01LA03","allCodes":["S01LA03"]},"biosimilarFilings":[],"originalDeveloper":"Valeant Pharms Llc","recentPublications":[{"date":"2006","pmid":"34813254","title":"Pegaptanib.","journal":""},{"date":"2003 May","pmid":"12714656","title":"VEGF164 is proinflammatory in the diabetic retina.","journal":"Investigative ophthalmology & visual science"},{"date":"2003 Mar","pmid":"12573989","title":"Intrauterine hypertension decreases lung VEGF expression and VEGF inhibition causes pulmonary hypertension in the ovine fetus.","journal":"American journal of physiology. Lung cellular and molecular physiology"},{"date":"2003 Jan","pmid":"12506087","title":"Controlled delivery of the anti-VEGF aptamer EYE001 with poly(lactic-co-glycolic)acid microspheres.","journal":"Investigative ophthalmology & visual science"},{"date":"2002 Apr","pmid":"11927845","title":"Preclinical and phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration.","journal":"Retina (Philadelphia, Pa.)"}],"companionDiagnostics":[],"genericManufacturerList":[],"status":"active","companyName":"Valeant Pharms Llc","companyId":"valeant-pharms-llc","modality":"Small molecule","firstApprovalDate":"2004","enrichmentLevel":3,"visitCount":0,"regulatoryByCountry":[{"country_code":"CA","regulator":"Health Canada","status":"approved","approval_date":null,"mah":"","brand_name_local":"","application_number":""},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null}],"trialStats":{"total":0,"withResults":0},"validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T03:31:57.682867+00:00","fieldsConflicting":1,"overallConfidence":0.8},"verificationStatus":"partial","dataCompleteness":{"mechanism":false,"indications":true,"safety":false,"trials":true,"score":2}}