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Pazopanib eye drops: a randomised trial in neovascular age-related macular degeneration
  1. Ronald Danis1,
  2. Megan M McLaughlin2,
  3. Michael Tolentino3,
  4. Giovanni Staurenghi4,
  5. Li Ye5,
  6. Chun-Fang Xu6,
  7. Robert Y Kim2,
  8. Mark W Johnson7,
  9. for the Pazopanib Eye Drops Study Group
  1. 1Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison School of Medicine and Public Health, Madison, Wisconsin, USA
  2. 2GlaxoSmithKline, King of Prussia, Pennsylvania, USA
  3. 3Center for Retina and Macular Disease, Winter Haven, Florida, USA
  4. 4Department of Clinical Science, Eye Clinic, University of Milan, Novara, Italy
  5. 5GlaxoSmithKline, Upper Providence, Pennsylvania, USA
  6. 6GlaxoSmithKline, Hertfordshire, UK
  7. 7Department of Ophthalmology and Visual Sciences, University of Michigan School of Medicine, Ann Arbor, Michigan, USA
  1. Correspondence to Dr Ronald Danis, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, School of Medicine and Public Health, 8010 Excelsior Drive, Madison, WI 53717, USA; rpdanis{at}


Aims To evaluate pazopanib eye drops in patients with subfoveal choroidal neovascularisation secondary to age-related macular degeneration.

Methods 70 patients with minimally classic or occult subfoveal choroidal neovascularisation were randomly assigned to 5 mg/mL TID, 2 mg/mL TID, and 5 mg/mL QD pazopanib eye drops for 28 days in a multicentre, double-masked trial with an optional safety extension for up to 5 additional months. The primary outcomes were central retinal thickness (CRT) and best-corrected visual acuity (BCVA) at Day 29.

Results No significant decrease from baseline in CRT was observed overall; however, an exploratory analysis showed improvement in CRT (mean decrease of 89 μm) in patients with the CFH TT genotype who received 5 mg/mL TID (p=0.01, n=5). Mean increases in BCVA were observed in the 5 mg/mL TID overall (4.32 letters (p=0.002, n=26)) and in those that with CFH Y402H TT (6.96 letters (p=0.02, n=5)) and CT (4.09 letters (p=0.05, n=9)) genotypes. No safety signals that precluded continued investigation were detected.

Conclusions 5 mg/mL pazopanib eye drops resulted in mean improvement in BCVA at Day 29 and improvements in vision. However, improvement in macular oedema for age-related macular degeneration was found only in the subset of subjects with the CFH Y402H TT genotype, warranting further investigation.

  • Clinical Trial
  • Diagnostic tests/Investigation
  • Drugs
  • Macula
  • Neovascularisation

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