A daily 15 mg dose of the oral medication pazopanib for patients with age-related macular degeneration (AMD, a leading cause of blindness) resulted in improvements in vision and other measures related to the retina, according to a report of two clinical trials by Megan M. McLaughlin, M.S., of GlaxoSmithKline, King of Prussia, Pa., and colleagues.
Most of the vision loss caused by AMD is connected to new blood vessel formation in the retina, according to the study background. Treatment with a drug that blocks new blood vessel formation is effective but must be given by injection in the eye. Pazopanib is a new drug that blocks blood vessel formation that can be given by mouth.
The clinical trials included 72 healthy controls and 15 patients with AMD. Healthy participants were administered 5 to 30 mg of oral pazopanib daily and patients with AMD were given 15 mg daily.
According to the findings, oral pazopanib (15 mg daily) was well tolerated and resulted in improvements in vision, according to the study.
"Based on safety and preliminary efficacy, these studies identified a dose of oral pazopanib that may be appropriate for further investigation in patients with neovascular AMD," the authors conclude. This was a phase 1 trial intended to establish a dose with acceptable efficacy and safety that could be studied more intensively.