Regeneron Pharmaceuticals (NASDAQ:REGN) saw its shares rise Monday after it announced over the weekend that the FDA has approved its Eylea injection treatment for wet age-related macular degeneration (AMD), a leading cause of acquired blindness.
The treatment, known in the scientific literature as VEGF Trap-Eye, was approved at a recommended dose of 2 milligrams every four weeks for the first 12 weeks, followed by 2 milligram dose every two months.
Vascular Endothelial Growth Factor (VEGF) is a naturally-occurring protein in the body, and its normal role is to trigger formation of new blood vessels, supporting the growth of the body's tissues and organs. However, in certain diseases, such as wet age-related macular degeneration, it is also associated with the growth of abnormal new blood vessels in the eye, which results in scarring, and loss of central vision.
Eylea works to inhibit the binding and activation of VEGF receptors.
The approval of Eylea was granted under a priority review, a designation that is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy currently exists.
The green light was based upon the results of two phase three studies, Regeneron said, which showed that Eylea, dosed every eight weeks following three initial montly injections, was "clinically equivalent" to standard of care treatment Lucentis dosed every four weeks.
The primary goal of the studies was the proportion of patients who maintained vision, defined as losing fewer than 15 letters of vision loss on an eye chart, over 52 weeks.
"The approval of Eylea offers a much needed new treatment option for patients with wet AMD," said the chair of the steering committee for the phase three trial, Jeffrey Heier, M.D., also a clinical ophthalmologist and retinal specialist at Ophthalmic Consultants of Boston, and Assistant Professor of Ophthalmology at Tufts School of Medicine.
"Eylea offers the potential of achieving the efficacy we've come to expect from current anti-VEGF agents, but with less frequent injections and no monitoring requirements. This may reduce the need for costly and time-consuming monthly office visits for patients and their caregivers."
President and CEO of Regeneron, Leonard S. Schleifer, added: "This approval is an important step forward for Regeneron and for patients suffering with wet AMD, the most common cause of blindness in the U.S. in older adults."
Eylea is planned to be available to patients within the next few days, the company said. Regeneron is collaborating with Bayer on the global development of the drug, with Bayer having submitted an application for marketing authorization in Europe for wet AMD in June.
Bayer will market Eylea outside the US, where the companies will share the profits equally. Regeneron maintains exclusive rights to the drug in the US.
Macular degeneration is diagnosed as either dry or wet. In wet AMD, new blood vessels grow beneath the retina and leak blood and fluid, which causes disruption and dysfunction and creates distortion and/or blind spots in central vision. Wet AMD is the leading cause of blindness for people over the age of 65 in the U.S. and Europe.
Regeneron's shares rose nearly 12% to trade at $55.58 as of 2:29pm ET on Monday afternoon.