FDA approves EYLEA for treatment of retinopathy of prematurity


Aflibercept, a VEGF inhibitor previously indicated for a variety of retinal diseases, is the first pharmacological treatment for retinopathy of prematurity in preterm infants.

Preterm infant holds mother's hand

Today, Regeneron announced that EYLEA (aflibercept) injection was approved by the FDA for retinopathy of prematurity (ROP) in preterm infants. Aflibercept is the first pharmacological treatment to be approved by the FDA for ROP and is the first pediatric indication for EYLEA. With this new approval, EYLEA is now approved to treat 5 retinal diseases linked to 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 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.”

ROP affects between 1,100 and 1,500 preterm infants, generally born before 31 weeks of pregnancy and weighing less than 3.3 lbs (1,500 grams) in the US every year. Retinal blood vessels develop when the infant is full-term (around 9 months of pregnancy), thus preterm births increase the risk of abnormal blood vessel development (retinal neovascularization) and possibly retinal detachment and irreversible vision loss. While mild cases of ROP may improve without pharmaceutical intervention, moderate to severe cases need treatment to prevent visual impairment or vision loss.

“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,” Jeff Todd, Chief Executive Officer of Prevent Blindness, said 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.”

About the ROP phase 3 program

This FDA approval comes supported by data from two randomized global phase 3 trials—FIREFLEYE and BUTTERFLEYE—which evaluated EYLEA 0.4mg against laser photocoagulation (laser) in infants with ROP. Around 80% of EYLEA-treated infants between the two trials reached an absence of active ROP, as well as the absence of unfavorable structural outcomes at 52 weeks of age. These results are better than expected without treatment, and 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. Serious ocular AEs occurred in 8% for both groups in FIREFLEYE and 6.5% versus 11% in BUTTERFLEYE. AEs in both phase 2 trials were consistent with infant prematurity or to the injection procedure, and saw AEs consistent with similar ROP trials.

The results of FIREFLEYE were published in the Journal of the American Medical Association, and data from BUTTERFLEYE were presented at the ROP Update 2022 meeting held in the United States. Both respective trials were conducted pursuant to FDA Pediatric Written Request, receiving a Pediatric Exclusivity Determination granting by the FDA in October 2022. This grant extends the US market exclusivity for EYLEA by an additional six months through May 17, 2024.

Disclosures: 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 US. Bayer has licensed the exclusive marketing rights outside of the US, where the companies share equally the profits from EYLEA sales.

1. EYLEA® (aflibercept) injection approved as the first pharmacologic treatment for preterm infants with retinopathy of prematurity (ROP) by the FDA. Press release. Regeneron; February 8, 2023. Accessed February 9, 2023. https://investor.regeneron.com/news-releases/news-release-details/eylear-aflibercept-injection-approved-first-pharmacologic
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