Approval of Macugen as a treatment for age-related macular degeneration (AMD) "represents the beginning of a new era," according to Judah Folkman, MD, who first wrote about such anti-angiogenic treatments for AMD 34 years ago

"Macugen is the first anti-angiogenic treatment approved in ophthalmology, said Folkman, who is Julia Andrus Dyckman professor of pediatric surgery at Children's Hospital and Harvard Medical School (both Boston), noting that such therapy had evolved from "theory to therapy."

Folkman first wrote about such anti-angiogenic treatments for AMD 34 years ago in the New England Journal of Medicine and said he supports the FDA's decision, which came in late December.

Macugen (pegaptanib sodium injection) is produced by Eyetech Pharmaceuticals (New York), which has a marketing partnership with Pfizer (New York). The potential blockbuster treatment for neovascular, or wet, AMD is expected to compete head-to-head with Visudyne, a treatment for the predominate form of wet AMD.

Eyetech CEO David Guyer said in a conference call at the time of the approval that 15 million people in the U.S. are living with some form of macular degeneration. Of those, 1.6 million are experiencing the active blood vessel growth and blood vessel leakage in the eye that is associated with neovascular AMD, which frequently leads to blindness.

"The FDA's approval of Macugen . . . represents an important paradigm shift in the treatment of this devastating disease," Guyer said. "I think Macugen is a novel treatment based on elegant science that for the first time targets the underlying cause of the disease, which led to our broad AMD label, including all lesion subtypes and lesions."

Guyer described Macugen as "the first in a new class of ophthalmic drugs to specifically target vascular endothelial growth factor, or VEGF."

The AMD Alliance International (Toronto) said it "welcomes" the FDA's decision to approve Macugen. "This decision is very timely. Wet AMD is a devastating disease that places a tremendous burden on patients and their families because it robs people of their central vision, making it difficult to lead independent lives," said Wanda Hamilton, executive director of the alliance.

The company's new drug application was based on findings from two pivotal Phase II/III randomized, multicenter, double-masked clinical trials involving about 1,200 patients with all subtypes of neovascular AMD. Results showed that among patients receiving 0.3 mg of Macugen, 70% lost less than three lines of vision on the eye chart, compared with 55% of patients receiving control treatment.

What's more, Guyer said, those patients receiving Macugen after two years fared better than those patients who only received the drug for one year, something he said points to the chronic nature of AMD.

He said that Eyetech would have a dedicated retinal sales force for Macugen that will promote the treatment to its target audience, which includes about 1,400 retinal specialists. Pfizer will be contributing 150 sales representatives to Macugen who will be targeting general ophthalmologists and optometrists.

Karen Young