Eylea Approved as First Pharmacologic Treatment of Preterm Infants with ROP
The FDA has approved Eylea (aflibercept) 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,” George D. Yancopoulos, MD, PhD, President and Chief Scientific Officer of Regeneron, and a principal inventor of Eylea, said in a company news release. “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.”
The FDA approval was 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 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.
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 US.
Each year in the US, 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 3.3 lbs pounds (1,500 grams) 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, Chief Executive Officer, 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.”
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.