FDA Approves Lucentis to Treat Diabetic Macular Edema

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For many people who have experienced diabetic macular edema (DME), the future looks a little clearer. On August 10, 2012, the U.S. Food and Drug Administration (FDA) approved use of the drug Lucentis for the treatment of DME. Developed by Genentech, Lucentis is the first and only FDA-approved medicine for the condition.

Diabetic macular edema is a complication of type 1 and type 2 diabetes and the leading cause of new blindness in people ages 20 to 74 in the United States. According to JDRF senior scientific program manager for complications therapies Helen Nickerson, Ph.D., “Recent international analysis showed that nearly 20 percent of people with 20 years of diabetes have DME.” Until now, the only standard treatment available was laser treatment. Such therapy, though it stalls the progression of the disease, does not reverse visual loss. For many people, Lucentis goes a significant step further.

Originally developed to treat age-related macular degeneration, Lucentis (ranibizumab injection) works by targeting vascular endothelial growth factor (VEGF), a protein whose function is to promote the growth of new blood vessels. In people with diabetic retinopathy, VEGF becomes overactive, and the new blood vessels can rupture and leak fluid, causing the retina to swell and affecting the macula, the part of the eye where color and fine detail are perceived. Lucentis is designed to inhibit VEGF, in turn limiting the growth of new blood vessels under the retina. Lucentis was approved to treat DME in Europe and Canada in 2011.

JDRF has directly supported key research in this area for more than 15 years, and has advocated for funding of trials through the National Institutes of Health’s (NIH) Special Diabetes Program. In the 1990s, JDRF supported multiple basic science grants that contributed to the knowledge that VEGF might be targeted to treat diabetic eye disease. From 2000, JDRF partnered with the NIH’s Diabetic Retinopathy Clinical Research (DRCR) Network and the National Eye Institute in a clinical trial that tested the short-term efficacy of the anti-VEGF bevacizumab (trade name Avastin) for DME, as well as a study testing whether Lucentis can improve treatment for proliferative diabetic retinopathy.

The results were encouragingly positive. The DRCR went on to perform a longer-term study comparing the efficacy of laser therapy, laser therapy with Lucentis, Lucentis alone, and steroid treatment for DME. That study showed that Lucentis treatment, with or without laser therapy, produced better vision outcomes than laser or steroid treatment. JDRF also collaborated with Genentech and the Wilmer Eye Institute of The Johns Hopkins University to test the safety and efficacy of Lucentis treatment alone. The results of the pivotal Phase III studies conducted by Genentech showed that between 34 and 45 percent of study subjects who received monthly Lucentis injections gained at least three lines of vision on a standard eye chart, compared to 12 to 18 percent of control-group subjects, who did not receive Lucentis.

The drug-development process does not end with clinical trials, however—and neither does JDRF’s work to advance new treatments for people with T1D. On July 26 Dr. Nickerson provided a statement during review of Lucentis by the FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee (DODAC) meeting.

“The number and proportion of people in the U.S. and the world who will develop diabetic retinopathy over the next two decades will grow enormously,” Dr. Nickerson said. “We believe, therefore, that there is an urgent need for new therapies.” The DODAC voted in favor of approval, and on August 10, the FDA granted approval of Lucentis for treatment of DME.

Physicians can now prescribe Lucentis for their patients with DME; the monthly injection can be administered by an ophthalmologist.