Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Eylea (aflibercept) Injection to treat preterm infants with retinopathy of prematurity (ROP). Following this first pediatric approval, EYLEA is now indicated to treat five retinal conditions caused by ocular angiogenesis.
“Retinopathy of prematurity is a leading cause of childhood blindness worldwide. Until now, the only FDA-approved treatment in common use was laser photocoagulation, a complex and lengthy procedure that permanently ablates retina tissue and is stressful not only for infant patients but also the family navigating a delicate time after a preterm birth,” said George D. Yancopoulos, MD, PhD, president and chief scientific officer of Regeneron, and a principal inventor of Eylea. “For the first time, physicians will now have an FDA approved medication in EYLEA to treat this heartbreaking disease in these smallest of patients. We thank the investigators and the many families who participated in the clinical trials.”
Each year in the U.S., between 1,100 to 1,500 infants develop ROP that is severe enough to require medical treatment. This rare eye disease often impacts infants who are born before 31 weeks of pregnancy have been completed or who weigh less than 1,500 grams (3.3 lbs) pounds at birth.
As retinal blood vessels are often only fully developed once an infant is full-term (~9 months of pregnancy), these infants are at risk of developing retinal blood vessels that are abnormal (retinal neovascularization) potentially leading to retinal detachment and irreversible vision loss. Mild cases of ROP may improve without treatment, but some cases require treatment to keep ROP from causing significant visual impairment and even blindness.
“With no existing FDA approved guidance for the treatment of retinopathy of prematurity with anti-VEGF therapies, there was a significant need for research to understand how best to treat the disease in a manner that puts patient safety first and preserves vision for a lifetime,” said Jeff Todd, CEO, Prevent Blindness. “Regeneron’s trials investigating Eylea in retinopathy of prematurity have advanced our understanding of how to treat this disease and provided a needed evidence-based treatment option to potentially help preterm infants preserve their vision.”
About the phase 3 program in ROP
The FDA approval is supported by data from two randomized global Phase 3 trials – FIREFLEYE (N=113) and BUTTERFLEYE (N=120) – investigating EYLEA 0.4 mg versus laser photocoagulation (laser) in infants with ROP. In both trials, approximately 80% of EYLEA-treated infants achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age, which is better than would have been expected without treatment.
No new EYLEA safety signals were observed in either trial. Comparing EYLEA to laser, ocular adverse events (AEs) among patients occurred in 39% versus 37% in FIREFLEYE and 18% versus 26% in BUTTERFLEYE, with serious ocular AEs occurring in 8% for both groups in FIREFLEYE and 6.5% versus 11% in BUTTERFLEYE. AEs in both trials were consistent with infant prematurity or to the injection procedure, and with the AEs in similar ROP trials.
The results of FIREFLEYE were published in Journal of the American Medical Association, and data from BUTTERFLEYE were presented at ROP Update 2022 meeting in the U.S.
Both trials were conducted pursuant to FDA Pediatric Written Request, and a Pediatric Exclusivity Determination was granted by FDA on October 12, 2022. This grant extends the period of U.S. market exclusivity for Eylea by an additional six months through May 17, 2024.
EYLEA is being jointly developed by Regeneron and Bayer. The lead sponsors of the trials were Regeneron for BUTTERFLEYE and Bayer for FIREFLEYE. Bayer and Regeneron are collaborating on the global development of EYLEA. Regeneron maintains exclusive rights of EYLEA in the United States. Bayer has licensed the exclusive marketing rights outside the United States, where the companies share equally the profits from sales of Eylea.
About Eylea
EYLEA is a VEGF inhibitor formulated as an injection for the eye. It is designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels (vascular permeability) in the eye by blocking VEGF-A and placental growth factor (PLGF), two growth factors involved in ocular angiogenesis. The EYLEA safety and efficacy profile is supported by a robust body of research that includes eight pivotal Phase 3 trials, more than 11 years of real-world experience and greater than 57 million EYLEA injections globally.