First major treatment advance in more than 25 years for sight-threatening condition
The U.S. Food and Drug Administration announced Aug. 8 its approval of Lucentis (ranibizumab injection) for the treatment of diabetic macular edema, an eye condition in people with diabetes that causes blurred vision, severe vision loss and sometimes blindness.
Dr. David M. Brown, a retinal surgeon at The Methodist Hospital in Houston, Texas, and clinical associate professor of ophthalmology at Weill Cornell Medical College, was one of the lead investigators in the research that led to the Food and Drug Administration's approval. In addition, Dr. Donald J. D'Amico, professor and chairman of the Ophthalmology Department at Weill Cornell Medical College and ophthalmologist-in-chief at NewYork-Presbyterian Hospital/ Weill Cornell Medical Center and an expert on diabetic retinopathy, was invited by Genentech, Inc. to represent the community of retinal specialists in the United States. He gave a presentation "Diabetic Macular Edema: An Unmet Medical Need" on July 26 to the FDA advisory panel that recommended the drug for approval, urging the panel to consider diabetic macular edema as "an extremely important unmet medical need."
The drug is the first and only FDA-approved medicine for diabetic macular edema, a condition for which the standard of care has not changed significantly in more than 25 years. To date, the usual treatment in the United States has been laser surgery, which slows the rate of vision loss and helps stabilize vision, but has demonstrated only limited ability to restore lost vision.
"This approval is an important advancement in the fight against blindness for people with diabetes," Dr. Brown said. "Now that it will be available, Lucentis therapy can begin to make a difference in the lives of our patients with diabetic macular edema."
Nearly 26 million Americans have diabetes, which is the leading cause of new cases of blindness in adults aged 20 to 74. Diabetic macular edema is estimated to affect more than 560,000 Americans with the disease.
Diabetic macular edema is swelling of the macula, the central part of the retina responsible for sharp, central vision. The condition begins with diabetes, which can cause damage to blood vessels in the eye over time. When this happens, a patient is said to have diabetic retinopathy, the most common diabetic eye disease. The damaged blood vessels can leak blood and fluid, causing swelling and blurred vision, severe vision loss and sometimes blindness. Among Americans aged 40 years and older, more than 4.2 million have diabetic retinopathy.
Lucentis Efficacy in diabetic macular edema
The FDA first approved Lucentis for treatment of wet age-related macular degeneration in 2006 and for macular edema following retinal vein occlusion in 2010. Brown and his research team were integrally involved in the research that led to these FDA approvals as well.
The approval of Lucentis in diabetic macular edema was based on manufacturer Genentech's Phase III trials, RIDE and RISE, two identically-designed, parallel, double-masked, three-year clinical trials, which were sham-treatment controlled for 24 months. A total of 759 patients were randomized into three groups to receive monthly treatment with 0.3 mg Lucentis (n=250), 0.5 mg Lucentis (n=252) or sham injection (control group, n = 257). In the studies, treatment with Lucentis demonstrated improved clinical outcomes including substantial visual gain for many DME patients. Results showed patients who received 0.3 mg Lucentis experienced significant, early (Day 7) and sustained (24 months) improvements in vision.
More patients who received Lucentis were able to read at least three additional lines — 15 letters — on the eye chart at 24 months, had average vision gains exceeding two lines — 10 letters — on the eye chart at 24 months and were significantly more likely to maintain their vision—lose less than 15 letters on the eye chart — at 24 months.