Macugen

On December 20, 2004, the FDA authorized the first ophthalmic anti-angiogenic drug, Macugen, for the treatment of neovascular (wet) age-related macular degeneration. This drug, developed in concert by Eyetech Pharmaceuticals, Inc. and Pfizer, Inc., helps preserve vision and helps slow progression to legal blindness.

"Macugen represents an important paradigm shift in the treatment of this devastating disease," said Eyetech CEO David Guyer, noting that the theory behind the drug’s therapeutic approach has roots that date back 35 years. "It’s a novel treatment that’s based on elegant science."

Macugen (pegaptanib sodium injection) is the first in a new class of ophthalmic drugs to specifically target vascular endothelial growth factor (VEGF), a protein that acts as a signal in triggering the abnormal blood vessel growth and leakage that characterize wet age-related macular degeneration (AMD). "What’s very important about Macugen,” Guyer told BioWorld Today “is that it targets the underlying biology of the disease."

The drug is administered in a 0.3 mg dose once every 6 weeks by intravitreal injection (injected directly into the eye). Macugen is the first therapy indicated for the treatment of wet AMD, regardless of lesion subtype or size. Previously, the only FDA-approved treatment, Visudyne, was limited to the predominantly classic subtype of wet AMD. That accounts for about 25% of the wet AMD patient population, but it still generated $357 million in sales last year.

"From our perspective, the fact that the FDA has approved the all-wet AMD label is one way to assure that the patients have access to the drug,” Paul Chaney, Eyetech’s chief operation officer, told BioWorld Today. “And physicians know that they are being reimbursed for the delivery of the drug to the patients they feel are appropriate and can benefit from the treatment."

In the US, more than 1.6 million of the estimated 15 million AMD patients have the active blood vessel growth and blood vessel leakage associated with wet AMD. There are more than 200,000 cases of wet AMD diagnosed each year, and Eyetech and Pfizer expect that number to increase as baby-boomers age and overall life expectancy improves.

"The goal is to focus the majority of our efforts, from a product distribution and access perspective, to retinal specialists who really are at the center of AMD treatment," Chaney said. "We also want to reach out to the general ophthalmology and optometry practices, so they know there is a new treatment available to offer something that is effective and safe for their patients, and then direct them to retinal specialists, who will be the ones who really drive the use of Macugen."

Data from the pivotal trials underlying the approval, which involved about 1,200 with all subtypes of wet AMD, showed that 70% of Macugen-treated patients lost fewer than three lines of vision acuity on the eye chart by week 54, compared to 55% of control treated patients.

MAP, the Macugen Assistance Program and Pfizer, is available to help patients deal with insurance coverage. Information about enrollment in the program is available at 866.272.8838. For medical information about Macugen, call 800.438.1985 and select option #3.

- Aaron Lorenzo, BioWorld Online

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