Novartis to file for new Lucentis (ranibizumab) indication in retinopathy of prematurity (ROP), a rare disease in premature infants that often leads to blindness

Novartis International AG / Novartis to file for new Lucentis (ranibizumab) indication in retinopathy of prematurity (ROP), a rare disease in premature infants that often leads to blindness . Processed and transmitted by West Corporation. The issuer is solely responsible for the content of this announcement.

• In the Phase III RAINBOW study, despite marginally missing statistical significance for the primary endpoint of demonstrating superiority of Lucentis to laser surgery, Lucentis  was shown to be an efficacious, safe and well-tolerated treatment for infants with ROP [1]
   

Laser surgery, the current standard of care for ROP in infants, destroys diseased retinal tissue responsible for elevated vascular endothelial growth factor (VEGF) whereas Lucentis is an anti-VEGF that directly targets and reduces VEGF[2]
 

With 80% of patients achieving treatment success with 0.2mg Lucentis versus 66% with laser, these data are clinically relevant[1]
 

Novartis plans to file ex-US for a new indication in ROP to bring transformative treatment to premature infants facing severe vision loss - the first anti-VEGF product to seek an ROP indication

Basel, September 22, 2018 - Novartis today 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[2],[3]. 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[4],[5],[6].

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[1]. 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.