FDA Approves Aflibercept for Retinopathy of Prematurity Treatment


Announced by Regeneron, aflibercept injection is the first FDA-approved pharmacologic treatment for preterm infants with ROP.

FDA Approves Aflibercept to Treat Retinopathy of Prematurity

The US Food and Drug Administration (FDA) has approved aflibercept (EYLEA®) injection as the first pharmacologic treatment for preterm infants with retinopathy of prematurity (ROP).

Announced by Regeneron Pharmaceuticals in a release1, the pediatric approval marks the fifth indication for aflibercept to treat 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 in a statement.1 “For the first time, physicians will now have an FDA-approved medication in EYLEA to treat this heartbreaking disease in these smallest of patients.”

Between 1,100 and 1,500 infants develop ROP that is severe enough to require medical treatment each year in the United States. It can impact infants who are born before 31 weeks of pregnancy have been completed or who weigh less than 3.3 pounds at birth. Retinal blood vessels are often only fully developed once an infant is full-term and pre-term infants are at risk of developing retinal neovascularization, which can lead to retinal detachment and irreversible vision loss.

Aflibercept is a vascular endothelial growth factor (VEGF) inhibitor formulated as an injection for the eye. The therapy is designed to block the growth of new blood vessels and decrease vascular permeability by blocking VEGF-A and placental growth factor.

The current approval was supported by data from two randomized global Phase 3 trials, the FIREFLEYE (n = 113) and BUTTERFLEYE (n = 120) investigating aflibercept 0.4 mg versus laser photocoagulation in infants with ROP. Results from both trials suggest approximately 80% of aflibercept-treated infants achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age. The release noted this as a better result than what is expected without treatment.

No new aflibercept safety signals were observed in either trial. Ocular adverse events were reported in 39% of aflibercept-treated patients versus 37% of laser-treated patients in FIREFLEYE and 18% of aflibercept-treated patients versus 26% of laser-treated patients in BUTTERFLEYE.

Serious ocular adverse events occurred in 8% of both groups in FIREFLEYE and 6.5% versus 11% in BUTTERFLEYE. Otherwise, adverse events in both trials were consistent with infant prematurity or to the injection procedure, and with the adverse events in similar ROP trials.

“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 in a statement.1 “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.”


  1. Eylea® (AFLIBERCEPT) injection approved as the first pharmacologic treatment for preterm infants with retinopathy of prematurity (ROP) by the FDA. Regeneron Pharmaceuticals Inc. https://investor.regeneron.com/news-releases/news-release-details/eylear-aflibercept-injection-approved-first-pharmacologic. Published February 8, 2023.

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