FDA Approves Aflibercept for Infants With Retinopathy of Prematurity


Aflibercept injection is also indicted for the treatment of neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy.

The FDA has approved aflibercept (Eylea; Regeneron Pharmaceuticals, Inc and Bayer) for the treatment of preterm infants with retinopathy of prematurity (ROP). This marks the first pediatric approval for aflibercept, which is now indicated to treat 5 retinal conditions caused by ocular angiogenesis. Aflibercept injection 2 mg is also indicted for the treatment of patients with neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy.

“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 principal inventor of aflibercept George D. Yancopoulos, MD, PhD, president and chief scientific officer of Regeneron, in a press 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 approval was based on data from the randomized, global FIREFLEYE (N=113) and BUTTERFLEYE (N=120) phase 3 trials, which evaluated aflibercept 0.4 mg compared with laser photocoagulation in infants with ROP. Across both studies, approximately 80% of infants administered aflibercept achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age, which is superior to anticipated outcomes without treatment, according to the investigators.

There were no new safety signals reported in either trial. Ocular adverse events (AEs) occurred in 39% of patients administered aflibercept compared with 37% in the laser cohort in FIREFLEYE and 18% versus 26%, respectively, in the BUTTERFLEYE trial. Serious ocular AEs were reported in 8% of both cohorts in FIREFLEYE and 6.5% versus 11%, respectively, in BUTTERFLEYE.

Aflibercept is a VEGF inhibitor designed to inhibit the growth of new blood vessels and reduce the ability of fluid to pass through blood vessels in the eye by blocking VEGF-A and placental growth factor, which are involved in ocular angiogenesis.

According to Regeneron, between 1100 and 1500 infants develop ROP that is severe enough to require medical treatment each year in the United States. ROP typically manifests in infants born before the completion of 31 weeks of pregnancy or in those who weigh less than 3.3 lbs pounds at birth.

Because retinal blood vessels typically only reach full development at full-term (~9 months of pregnancy), infants with ROP are at risk of developing retinal blood vessels that are abnormal (retinal neovascularization), which can lead to retinal detachment and irreversible vision loss. Mild cases of the condition may improve without treatment; however, some cases require intervention tp prevent ROP from causing significant visual impairment or 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 of Prevent Blindness, in a press release. “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® (Aflibercept) Injection Approved as the First Pharmacologic Treatment For Preterm Infants With Retinopathy of Prematurity (ROP) by the FDA. News release. Regeneron. February 8, 2023. https://investor.regeneron.com/news-releases/news-release-details/eylear-aflibercept-injection-approved-first-pharmacologic

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