Novartis to Seek Approval for Lucentis as Treatment for Rare Eye Disease

Rare Daily Staff

Novartis said it will seek approval to expand the indicated use of Lucentis outside of the United States to include retinopathy of prematurity (ROP), a rare disease that is the leading cause of childhood blindness.

ROP is caused by abnormal development of retinal blood vessels in premature babies. The retina is the inner layer of the eye that receives light and turns it into visual messages that are sent to the brain. The retina develops late in the womb and very premature babies may have incomplete development of blood vessels needed to provide oxygen.

Vascular Endothelial Cell Growth Factor (VEGF) is an important regulator of the development of new blood vessels (known as angiogenesis) and plays a key role in the progression of ROP. If the blood vessels do not develop normally during ROP, they can exert traction on the retina and lead to macular dragging, retinal detachment or other structural abnormalities resulting in vision loss or potentially blindness.

Lucentis is the first anti-VEGF therapy licensed for ophthalmic use. It has been used to treat neovascular or wet age-related macular degeneration, the leading cause of blindness, as well as other indications. Lucentis was developed by Genentech and Novartis.

The company said that results from a phase 3 (RAINBOW) study of Lucentis versus laser surgery, the current standard of care for ROP, marginally missed statistical significance for the primary endpoint but believes the results are nevertheless clinically significant. The company reported on results at the 18th Congress of the European Society of Retina Specialists.

Unlike laser surgery, which damages eye tissue and can be associated with significant complications such as high myopia, Lucentis targets and reduces the elevated intraocular level of VEGF, which is the underlying cause of the condition.

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,” said Andreas Stahl, senior physician in retinal surgery and head of the Angiogenesis Research Group at the Eye Center, University of Freiburg, Germany. “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.”

September 24, 2018
Photo: Andreas Stahl, senior physician in retinal surgery and head of the Angiogenesis Research Group at the Eye Center, University of Freiburg, Germany