The US Food & Drug Administration (FDA) has approved Roche's Lucentis (ranibizumab injection) for treatment of diabetic macular edema (DME), an eye condition in people with diabetes that causes blurred vision, severe vision loss and sometimes blindness.

Diabetes is now the leading cause of new cases of blindness in American adults, and DME is estimated to affect more than 560,000 Americans with the disease.

Lucentis is the first and only FDA-approved medicine for DME, a condition for which the standard of care has not changed significantly in more than 25 years. To date, the standard of care in the US for DME 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.

“For the first time, Americans with diabetic macular edema will have access to an FDA-approved medicine shown to help many patients rapidly regain substantial amounts of lost vision,” said Hal Barron, MD, chief medical officer and head, Global Product Development. “We developed Lucentis to treat diseases of the eye and are pleased to have received this third US indication to help a new population of people whose eyesight may be affected by diabetes.”

“This approval is an important advancement in the fight against blindness for people with diabetes,” said David M Brown MD, Retinal Specialist at The Methodist Hospital, Houston Texas, and clinical trial investigator. “Now that it will be available, Lucentis therapy can begin to make a difference in the lives of our patients with DME.”

Lucentis 0.5 mg once monthly was first approved by the FDA for treatment of wet age-related macular degeneration (AMD) in 2006 and for macular edema following retinal vein occlusion (RVO) in 2010. Lucentis 0.3 mg once monthly was approved for DME, and physicians can order immediately with shipments expected to begin August 15.

The approval of Lucentis in DME was based on 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). Primary outcomes were evaluated at 24 months and have been published in Ophthalmology.

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: RIDE: 34 percent in the 0.3 mg group versus 12 percent in the control group; RISE: 45 percent, 0.3 mg versus 18 percent, control (primary endpoint).

Patients who received Lucentis had average vision gains exceeding two lines (10 letters) on the eye chart at 24 months: RIDE: 10.9 letters, 0.3 mg versus 2.3 letters, control; RISE: 12.5 letters, 0.3 mg versus 2.6 letters, control.

Significant gains in average vision were observed seven days after the first treatment.

Patients who received Lucentis were significantly more likely to maintain their vision (lose < 15 letters on the eye chart) at 24 months: RIDE: 98 percent, 0.3 mg versus 92 per cent, control; RISE: 98 per cent, 0.3 mg versus 90 per cent, control.

For all time points comparing 0.3 mg Lucentis to control through month 24 p < 0.01. Vision improvements observed in patients treated with Lucentis at 24 months were maintained with continued treatment through 36 months.

Lucentis is a prescription medicine for the treatment of patients with wet AMD, macular edema following RVO, and DME. Lucentis is a recombinant humanized monoclonal antibody fragment (lacking a Fc region). Lucentis is the first VEGF inhibitor specifically designed for use in the eye to bind to and inhibit VEGF-A, a protein that is believed to play a critical role in the formation of new blood vessels (angiogenesis) and the hyperpermeability (leakiness) of the vessels.

In wet AMD, these new blood vessels grow under the retina and leak blood and fluid, causing rapid damage to the macula. Lucentis administered monthly in wet AMD clinical trials demonstrated an improvement in vision of three lines or more on the study eye chart in up to 41 per cent of patients at two years. Nearly all patients (90 per cent) treated monthly with Lucentis in those trials maintained (defined as losing < 15 letters) vision.

In RVO, angiogenesis and hyperpermeability can lead to macular edema, the swelling and thickening of the macula. Lucentis administered at 0.5 mg monthly in RVO clinical trials demonstrated the following average vision gains for patients at six months: Patients with branch-RVO experienced an average gain of 18.3 letters on the study eye chart (compared to 7.3 letters for the control group) and patients with central-RVO experienced an average gain of 14.9 letters on the study eye chart (compared to 0.8 letters for the control group).

Lucentis has been rigorously studied in multiple retinal diseases in 27 clinical trials involving more than 10,500 patients worldwide. Outside the US, Lucentis has received regulatory approval for treatment of visual impairment due to DME in more than 75 countries, for treatment of wet AMD in more than 100 countries and for treatment of RVO in more than 70 countries.

Lucentis was discovered by Genentech and is being developed by Genentech and Novartis for diseases or disorders of the eye. Genentech retains commercial rights in the US and Novartis has exclusive commercial rights for the rest of the world.

Roche, the world’s largest biotech company and is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics.