A Simpler Treatment for Macular Degeneration

Oral pazopinab for age related macular degeneration was found to be safe and effective

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

(RxWiki News) Typical treatment options for age-related macular degeneration — an eye condition that leads to vision loss in older people — can be invasive and uncomfortable, but there may be a simpler way to treat this disease.

A recent study found that the medication Votrient (pazopanib), which is used in the treatment of certain cancers, was safe and effective for patients with age-related macular degeneration (AMD).

Due to the small study sample, the researchers noted that more studies are needed to determine if Votrient is appropriate for all AMD patients or only a specific group.

"Speak with your doctor about treatment options for your eye condition."

This research was led by Megan M. McLaughlin, MS, with GlaxoSmithKline in King of Prussia, Pennsylvania. The research team evaluated the safety of oral pazopanib in a group of healthy participants, and they also evaluated its safety and effectiveness in treating AMD for patients with that condition.

AMD typically occurs in people over the age of 50, and happens when the part of the eye that is used for central vision begins to break down.

This phase I trial had two parts.

The first part of the study included 72 participants who were healthy and at least 18 years of age. These healthy participants were only studied to determine the safety of oral pazopanib and for researchers to learn how the drug was processed in the body. 

The second part of the study included 15 patients who had AMD, were at least 50 years of age and had never previously taken oral pazopanib. The information from this second part was used to determine how effective oral pazopanib was in treating AMD.

Healthy participants (from the first part of the study) received one dose of 5 to 30 milligrams of oral pazopanib, multiple doses of 5 to 30 milligrams of oral pazopanib or a placebo (fake medication) once per day for 14 days. AMD patients (from the second part of the study) received 15 milligrams of oral pazopanib once daily for 28 days.

The researchers specifically examined the safety and tolerability of oral pazopanib as well as its effect on visual outcomes, including visual acuity (clearness of vision) and effects on the retina.

Nine out of the 15 AMD patients were able to complete the study without needing rescue treatment (standard of care treatment for AMD). The researchers also observed a small but significant improvement in some of the clinical signs of AMD, including visual acuity.

The adverse events in healthy participants included abdominal pain (at a dose of 10 milligrams), fatigue (at 20 milligram and 30 milligram doses) and headache (at doses of 10 milligrams and 30 milligrams).

Seven out of 15 patients reported experiencing a mild to moderate eye-related adverse event such as retinal hemorrhage or retinal edema.

As the researchers noted, AMD is the number one cause of newly developed blindness in people aged 65 years and older. Typical treatments for AMD, such as repeated eye injections and laser therapy, can be invasive. Oral pazopanib could serve as a simpler way to effectively treat this disease.

These findings offer a starting point for determining a safe and recommended dose of oral pazopanib in treating patients with AMD. Future phases of this research will work to identify a specific, suitable dose for AMD patients.

The researchers concluded that their findings demonstrated that oral pazopanib was generally safe, well tolerated and effective for AMD patients taking 15 milligrams per day for 28 days.

This study was published on October 10 in JAMA Ophthalmology.

This study was funded by GlaxoSmithKline.

Some of the study authors reported competing interests with GlaxoSmithKline, the pharmaceutical company that manufactures oral pazopanib.

Review Date: 
October 10, 2013
Last Updated:
October 14, 2013