Novartis reports Phase III study of Lucentis to treat retinopathy of prematurity.

Novartis has announced results from a Phase III study of Lucentis (ranibizumab) versus laser surgery (the current standard of care) at the 18th Congress of the European Society of Retina Specialists (EURETINA) in premature infants with retinopathy of prematurity (ROP), a rare disease but a leading cause of childhood blindness.

Unlike laser surgery, which damages eye tissue and can be associated with significant complications such as high myopia, Lucentis pharmacologically targets and reduces the elevated intraocular level of vascular endothelial growth factor (VEGF), which is the underlying cause of the condition. Although the RAINBOW Phase III study marginally missed statistical significance for the primary endpoint (p=0.0254, as opposed to the significance level of p=0.025), the difference in treatment success between the Lucentis 0.2mg and 0.1mg groups compared to laser surgery (80%, 75% and 66.2% respectively) is nevertheless considered clinically relevant by Novartis. Based on the favorable benefit-risk profile, Novartis plans to file for a new indication for Lucentis for the treatment of ROP to support this rare but important patient population.

"Laser surgery, the current standard of care, works by destroying the tissue in the eye that contributes to the elevation of VEGF. While it is an effective treatment, there is a clear unmet need for innovative ways to treat ROP without destroying retinal tissue. Lucentis demonstrated in the RAINBOW study that it is an efficacious and well tolerated option for the treatment of ROP that may offer new hope to parents of this vulnerable patient population," said Professor Andreas Stahl, Senior Physician in Retinal Surgery and Head of the Angiogenesis Research Group at the Eye Center, University of Freiburg, Germany.

Retinopathy of prematurity affects premature infants in both developed and developing countries, with an estimated 23,800 to 45,600 infants newly diagnosed with irreversible vision impairment from ROP each year. Caused by the abnormal development of retinal blood vessels in premature infants, disease progression is due to high levels of a growth factor called VEGF. After premature birth, high VEGF levels can cause an infant's blood vessels in the retina to develop abnormally, which may lead to structural abnormalities such as retinal detachment, resulting in vision loss or blindness.