Pegaptanib sodium for the treatment of age-related macular degeneration

Expert Opinion on Pharmacotherapy

Volumn 9, Issue 3, 2008, Pages 499-508

  Apte, Rajendra S ^a  

^a WASHINGTON UNIVERSITY   (United States)

Author keywords

Age related macular degeneration; Aptamer; Choroidal neovascularization; Pegaptanib; Vascular endothdial growth factor

Indexed keywords

BENZOPORPHYRIN DERIVATIVE; BEVACIZUMAB; PEGAPTANIB; RANIBIZUMAB; RECOMBINANT PROTEIN; VASCULOTROPIN; VASCULOTROPIN INHIBITOR; ANGIOGENESIS INHIBITOR; APTAMER; ISOPROTEIN; VASCULOTROPIN A;

BLEEDING; CHEMISTRY; CLINICAL TRIAL; DIABETIC RETINOPATHY; DISEASE MODEL; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG METABOLISM; DRUG SAFETY; DRUG STABILITY; DRUG STRUCTURE; DRUG TOLERABILITY; ENDOPHTHALMITIS; FETUS MORTALITY; HUMAN; HYPERTENSION; NONHUMAN; PHARMACODYNAMICS; PHOTODYNAMIC THERAPY; PROTEINURIA; RETINA DETACHMENT; RETINA MACULA AGE RELATED DEGENERATION; RETINA VEIN OCCLUSION; REVIEW; SINGLE DRUG DOSE; SUBRETINAL NEOVASCULARIZATION; TERATOGENICITY; THROMBOEMBOLISM; TRAUMATIC CATARACT; VISUAL ACUITY; AGED; DRUG ANTAGONISM; RETINA MACULA DEGENERATION;

AGED; ANGIOGENESIS INHIBITORS; APTAMERS, NUCLEOTIDE; CHOROIDAL NEOVASCULARIZATION; HUMANS; MACULAR DEGENERATION; PROTEIN ISOFORMS; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 39049142223     PISSN: 14656566     EISSN: None     Source Type: Journal    
DOI: 10.1517/14656566.9.3.499     Document Type: Review

References (85)

1. National Eye Institute (2002). Available fiom: http://www.preventhrindness.org/vpus/amd.pdf
2. Resnikoff S, Pascolini D, Etya'ale D, et al. Global data on visual impairment in the year 2002. Bull World Health Organ 2004;82(11):844-51
3. Ferris FL 3rd, Fine SL, Hyman L Age-related macular degeneration and blindness due to neovascular maculopathy. Arch Ophthalmol 1984;102(11):1640-2
4. Ambati J, Ambati BK, Yoo SH, Ianchulev S, Adamis AP. Age-related macular degeneration: etiology, pathogenesis, and therapeutic strategies. Surv Ophthalmol 2003;48(3):257-93
5. Bermig J, Tylla H, Jochmann C, Nestler A, Wolf S. Angiographic findings in patients with exudative age-related macular degeneration. Graefes Arch Clin Exp Ophthalmol 2002;240(3):169-75
6. Comer GM, Ciulla TA, Criswell MH, Tolentino M. Current and future treatment options for nonexudative and exudative age-relared macular degeneration. Drugs Aging 2004;21(15):967-92
7. Macular Photocoagulation Study Group. Laser photocoagulation of subfoveal neovascular lesions in age-related macular degeneration. Results of a randomized clinical trial. Macular Photocoagulation Study Group. Arch Ophthalmol 1991;109(9):1220-31
8. TAP Study Group. Photodynamic therapy of subfoveal choroidal neovascalarization in age-related macular degeneration with verteporfin: one-year results of 2 randomized clinical trials -TAP report. Treatment of age-related macular degeneration with photodynamic therapy (TAP) Study Group. Arch Ophthalmol 1999;117(10):1329-45
9. Azab M, Benchaboune M, Blinder KJ, et al. Verteporfin therapy of subfoveal choroidal neovascularization in age-related macular degeneration: meta-analysis of 2-year safety results in three randomized clinical trials: Treatment Of Age-Related Macular Degeneration with Photodynamic Therapy and Verteporfin in Photodynamic Therapy Study Report no 4. Retina 2004;24(1):1-12
10. Leung DW, Cachianes G, Kuang VJ, Goeddel DV, Ferrara N. Vascular endothelial growth factor is a secreted angiogenic mitogen. Science 1989;246(4935):1306-9
11. Senger DR, Galli SJ, Dvorak AM, et al. Tumor cells secrete a vascular permeability factor that promotes accumulation of ascites fluid. Science 1983;219(4587):983-5
12. Kim I, Ryan AM, Rohan R, et al. Constitutive expression of VEGE VEGFR-1, and VEGFR-2 in normal eyes. Invest Ophthalmol Vis Sci 1999;40(9):2115-21
13. Ruckman J, Green IS, Beeson J, et al. 2-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165). Inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain. J Biol Chem 1998;273(32):20556-67
14. Ferrara N, Damico L, Shams N, Lowman H, Kim R. Development of ranibizumab, an anti-vascular endothelial growth factor antigen binding fragment, as therapy for neovascular age-related macular degeneration. Retina 2006;26(8):859-70
15. Lynch SS, Cheng CM. Beracizumab for neovascular ocular diseases. Ann Pharmacother 2007;41(4):614-25
16. Nguyen QD, Shah SM, Hafiz G, et al. A Phase I trial of an IV-administered vascular endothelial growth factor trap for treatment in patients with choroidal neovascularization due to age-related macular degeneration. Ophthalmology 2006;113(9):1522, e1521-2, e1514
17. Regeneron website (2001). Available from: http://www.regeneron.com/ company/press_detail.asp?v_c_id=283
18. Ferrara N. Vascular endothelial growth factor: basic science and clinical progress. Endocr Rey 2004;25(4):581-611
19. Adamis AP, Shima DT, Yeo KT, et al. Synthesis and secretion of vascular permeability factor/vascular endothelial growth factor by human retinal pigment epithelial cells. Biochem Biophys Res Commun 1993;193(2):631-8
20. Famiglietti EV, Stopa EG, McGookin ED, et al. Immunocytochemical localization of vascular endothelial growth factor in neurons and glial cells of human retina. Brain Res 2003;969(1-2):195-204
21. Aiello LP, Northrup JM, Keyt BA. Takagi H, Iwamoto MA. Hypooxic regulation of vascular endothelial growth factor in retinal cells. Arch Ophthalmol 1995;113(12):1538-44
22. Tolentino MJ, Miller JW, Gragoudas ES, et al. Vascular endothelial growth factor is sufficient to produce iris neovascularization and neovascular glaucoma in a nonhuman primate. Arch Ophthatmol 1996;114(8):964-70
23. Tolentino MJ, Miller JW, Gragoudas ES, et al. Intravitreous injections of vascular endothelial growth factor produce retinal ischemia and microangiopathy in an adult primate. Ophthalmology 1996;103(11):1820-8
24. Tolentino MJ, McLeod DS, Taomoto M, et al. Pathologic features of vascular endothelial growth for-induced retinopathy in the nonhuman primate. Am J Ophthalmol 2002;133(3):373-85
25. Spilsbury K, Garrett KL. Shen WY, Constable IJ, Rakoczy PE. Overexpression of vascular endothelial growth factor (VEGF) in the retinal pigment epithelium leads to the development of choroidal neovascularization. Am J Pathol 2000;157(1):135-44
26. Schwesinger C, Yee C, Rohan RM, et al. Intrachoroidal neovascularization in transgenic mice overexpressirig vascular endothelial growth factor in the retinal pigment epithelium. Am J Pathol 2001;158(3):1161-72
27. Ohno-Matsui Y, Hirose A, Yamamoto S, et al. Inducible expression of vascular endothelial growth factor in adult mice causes severe proliferative retinopathy and retinal detachment. Am J Pathol 2002;160(2):711-9
28. Aiello LP, Pierce EA, Foley ED, et al. Suppression of retinal neovascularization in vivo by inhibition of vascular endothelial growth factor (VEGF) using soluble VEGF-receptor chimeric proteins. Proc Natl Acad Sci USA 1995;92:10457-61
29. Adamis AP, Shima DT, Tolentino MJ, et al. Inhibition of vascular endothelial growth factor prevents retinal ischemia-associated. iris neovascularizadon in a nonhuman primate. Arch Ophthalmol 1996;114(1):66-71
30. Amano S. Rohan R, Kuroki M, Tolentino M, Adamis AP. Requirement for vascular endothelial growth factor in wound- and inflammation-related corneal neovascularization. Invest Ophthalmol Vis Sci 1998;39(1):18-22
31. Krzystolik MG, Afshari MA, Adamis AP, et al. Prevention of experimental choroidal neovascularization with intravitreal anti-vascular endothelial growth factor antibody fragment. Arch ophthalmol 2002;120(3):338-46
32. Robinson CJ, Stringer SE. The splice variants of vascular endothelial growth factor (VEGF) and their receptors. J Cell Sci 2001;114(Pt 5):853-65
33. Ishida S, Usui T, Yamashiro Y, et al. VEGF164-mediated inflammation is required for pathological, but not physiological, ischemia-induced retinal neovascularization. J Exp Med 2003;198(3):483-9
34. Ishida S, Usui T, Yamashiro K et al. VEGF164 is proinflammatory in the diabetic retina. Invest Ophthalmol Vis Sci 2003;44(5):2155-62
35. Usiti T, Ishida S, Yamashiro Y, et al. VEGF164(165) as the pathological isoform: differential leukocyte and endodidial responses through VEGFR1 and VEGFR2. Invest Ophthalmol Vis Sci 2004;45(2):368-74
36. Ng EW, Shima DT, Calias P, et al. Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Nat Rev Drug Discov 2006;5(2):123-32
37. Tuerk C, Gold L. Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science 1990;249(4968):505-10
38. Jellinek D, Green LS, Bell C, Janjic N. Inhibition of receptor binding by high-affinity RNA ligands to vascular endothelial growth factor. Biochemistry (Mosc) 1994;33(34):10450-6
39. Green LS, Jellinek D, Bell C, et al. Nuclease-resistant nucleic acid ligands to vascular permeability factor/vascular endothelial growth factor. Chem Biol 1995;2(10):683-95
40. Lee JH, Canny MD, De Erkenez A, et al. A therapeutic aptamer inhibits angiogenesis by specifically targeting the heparin binding domain of VEGF165. Proc Natl Acad Sci USA 2005;102(52):18902-7
41. Fairbrother WJ, Champe MA, Christinger HW, Keyt BA, Starovasnik MA. Solution structure of the heparin-binding domain of vascular endothelial growth Factor. Structure 1998;6(5):637-48
42. Tucker CE, Chen LS, Judkins MB, et al. Detection and plasma pharmacokinetics of an anti-vascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys. J Chromatogr B Biomed Sci Appi 1999;732(1):203-12
43. Bell C, Lynam E, Landfair DJ, Janjic N, Wiles ME. Oligonucleotide NX1838 inhibits VEGF 165-mediated cellular responses in vitro. In Vitro Cell Dev Biol Anim 1999;35(9):533-42
44. Eyetech Study Group. Preclinical and Phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration. Retina 2002;22(2):143-52
45. Drolet DW, Nelson J, Tucker CE, et al. Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys. Pharm Res 2000;17(12):1503-10
46. Pfizer website (2006). Available from: http://www.pfizer.com/pfizer/ download/uspi_macugen.pdf
47. Apte RS, Modi M, Masonson H, et al. Pegaptanib 1-year systemic safety results from a safety-pharmacokinetic trial in patients with neovascular age-related macular degeneration. Ophthalmology 2007;114(9):1702-12
48. Eyetech Study Group. Anti-vascular endothelial growth factor therapy for subfoveal choroidal neovascularization secondary to age-related macular degeneration: Phase II study results. Ophthalmology 2003;110(5):979-86
49. Gragoudas ES, Adamis AP, Cunningham ET Jr, Feinsod M, Guyer DR. Pegaptanib for neovascular age-related macular degeneration. N Engl J Med 2004;351(27):2805-16
50. Ng EW, Adamis AP. Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration. Can J Ophthalmol 2005;40(3):352-68
51. Chakravarthy U, Adamis AP, Cunningham ET Jr, et al. Year 2 efficacy results of 2 randomized controlled clinical trials of pegaptanib for neovascular age-related macular degeneration. Ophthalmology 2006;113(9):1508. e1501-25
52. Gonzales CR. Enhanced efficacy associated with early treatment of neovascular age-related macular degeneration with pegaptanib sodium: an exploratory analysis. Retina 2005;25(7):815-27
53. Ferrara. N, Mass RD, Campa C, Kim R. Targeting VEGF-A to treat cancer and age-related macular degeneration. Annu Rev Med 2007;58:491-504
54. Kamba T, McDonald DM. Mechanisms of adverse effects of anti-VEGF therapy for cancet. Br J Cancer 2007;96(12):1788-95
55. Verheul HM, Pinedo HM. Possible molecular mechanisms involved in the toxicity of angiogenesis inhibition. Nat Rev Cancer 2007;7(6):475-85
56. Blaauwgeers HG, Holtkamp GM, Rutten H, et al. Polarized vascular endothelial growth factor secretion by human retinal pigment epithelium and localization of vascular endothelial growth factor receptors on the inner choriocapillaris. Evidence fbr atrophic paracrine relation. Am J Pathol 1999;155(2):421-8
57. Marneros AG, Fan J, Yokoyama Y, et al. Vascular endothelial growth factor expression in the retinal pigment epithelium is essential for choriocapillaris development and visual function. Am J Pathol 2005;167(5):1451-9
58. Nishijima K, Ng YS, Zhong L, et al. Vascular endothelial growth Factor-A is a survival factor for retinal neurons and a critical neuroprotectant during the adaptive response to ischemic injury. Am J Pathol 2007;171(1):53-67
59. Genentech website (2006). Available firom: http://www.gene.com/gene/ products/information/oncology/avastin/insert.jsp#warnings
60. Demir R, Seval Y, Huppertz B. Vasculogenesis and angiogenesis in the early human placenta. Acta Histochem 2007;109(4):257-65
61. Brownbill P, McKeeman GC, Brockelsby JC, Crocker IP, Sibley CP. Vasoactive and permeability effects of vascular endothelial growth factor-165 in the term in vitro dually perfused human placental lobule. Endocrinology 2007;148(10):4734-44
62. Baumwell S, Karumanchi SA. Pre-eclampsia: clinical manifestations and molecular mechanisms. Nephron Clin Pract 2007;106(2):c72-81
63. D'Amico DJ, Masonson HN, Patel M, et al. Pegaptanib sodium for neovascular age-related macular degeneration: two-year safety results of the two prospective, multicenter, controlled clinical trials. Ophthalmology 2006;113(6):992-1001
64. Suner IJ. Safety of pegaptanib sodium in age-related macular degeneration (AMD): 3-year results of the VISION trial. Annual Meeting of the American Academy of Ophthalmology, Las Vegas, Nevada, 2006
65. Altaweel M; VISION Study Group. Effects of intravitreal injection of pegaptanib sodium (Macugen) on the retinal pigment epithelium and optic nerve. Invest Ophthalmol Vis Sci 2007;48:E-Abstiac 3369
66. Chang LK, Sarraf D. Tears of the retinal pigment epithelium: an old problem in a new era. Retina 2007;27(5):523-34
67. Friberg TR; LEVEL Study Group. Evaluation of efficacy and safety in maintaining visual acuity with sequential treatment of neovascular AMD: The LEVEL Study. Invest Ophthalmol Vis Sci 2007;48:E-Abstract 4568
68. Brown DM, Kaiser PK, Michels M, et al. Ranibizumab versus verteporfin for neovascular age-related macular degeneration. N Engl J Med 2006;355(14):1432-44
69. Rosenfeld PJ, Brown DM, Heier JS, et al. Ranibizumab for neovascular age-related macular degeneration. N Engl J Med 2006;355(14):1419-31
70. Bashshur ZF, Haddad ZA, Schakal A, Jaafar RF, Saab M, Noureddin BN. Intravitreal bevacizumab for treatment of neovascular age-related macular degeneration: a one-year prospective study. Am J Ophthalmol Epub 2007 Dec 5
71. La Cour M. Intravitreal VEGF-inhibitors: is Avastin a generic substitute for Lucentis? Acta Ophthalmol Scand 2007;85(1):2-4
72. Mitka M. Study aims to clarify efficacy; safety of eye drug treatments. JAMA 2007;297(14):1538-9
73. Van Wijngaarden P, Coster PJ, Williams KA. Inhibitors of ocular neovascularization: promises and potential problem. JAMA 2005;293(12):1509-13
74. Genentech website (2007). Available from: hnp://www.gene.com/gene/ products/information/pdf/healthcare-provider-letter.pdf
75. Gillies MC, Wong TY. Ranibizumab for neovascular age related macular degeneration. N Engl J Med 2007;356(7):748-9
76. Fung AE, Lalwani GA, Rosenfeld PJ, et al. An optical coherence tomography-guided, variable dosing regimen with intravitreal ranibizumab (Lucentis) for neovascular age-related macular degeneration. Am J Ophthalmol 2007;143(4):566-83
77. Hughes MS, Sang DN. Safety and efficacy of intravitreal bevacizumab followed by pegaptanib maintenance as a treatment regimen for age-related macular degeneration. Ophthalmic Surg Lasers Imaging 2006;37(6):446-54
78. Cunningham ET Jr, Adamis AP, Altaweel M, et al. A Phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema. Ophthalmology 2005;112(10):1747-57
79. Boyd SR, Zachary I, Chakravarthy U, et al. Correlation of increased vascular endothelial growth factor with neovascularization and permeability in ischemic central vein occlusion. Arch Ophthalmol 2002;120:1644-50
80. Adamis AP, Altaweel M, Bressler NM, et al. Changes in retinal neovascularization after pegaptanib (Macugen) therapy in diabetic individuals. Ophthalmology 2006;113:23-8
81. Gonzalez VH, Vann VR, Banda RM, et al. Pegaptanib sodium (Macugen®) versus panretinal photocoagulation (PRP) for the regression of proliferative diabetic retinopathy. Invest Ophthalmol Vis Sci 2007;48:E-Abstraa 4030
82. Wells JA III, Wroblewski JJ; The Pegaptanib in Retinal Central Vein Occlusion Study Group. Pegaptanib sodium for the treatment of macular edema following central retina vein occlusion (CRVO): functional outcomes. Invest Ophthalmol Vis Sci 2007;48:E-Abstract 1544
83. Filippopoulos T, Ducharme JF, Lowenstein JI, Krzystolik MG. Antiangiogenic agents as an adjunctive treatment for complicated neovascular glaucoma. Invest Ophthalmol Vis Sci 2006;47:E-Abstract 4476
84. Trese MT, Capone A Jr, Drenser K. Macugen in retinopathy of prematurity. Invest Ophthatmol Vis Sci 2006;47:E-Abstract 2330
85. Drenser KA, Trese MT, Capone A Jr. Macugen therapy for the treatment of familial exudative vitreoretinopathy. Invest Ophthalmol Vis Sci 2006;47-E-Abstract 4496