Eylea Approved for Preterm Infants With Retinopathy of Prematurity

The Food and Drug Administration (FDA) has approved Eylea^® (aflibercept) for the treatment of preterm infants with retinopathy of prematurity (ROP).

Eylea is a vascular endothelial growth factor (VEGF) inhibitor. The approval was based on data from the phase 3 BUTTERFLEYE (ClinicalTrials.gov Identifier: NCT04101721) and FIREFLEYE (ClinicalTrials.gov Identifier: NCT04004208) trials. Study participants had a maximum gestational age at birth of 32 weeks or a maximum birth weight of 1500g, had to weigh at least 800g on the day of treatment and had treatment-naïve ROP.

Patients were randomly assigned to receive 0.4mg of aflibercept via intravitreal injection or laser photocoagulation (the standard treatment for ROP). The primary endpoint was the proportion of patients with absence of active ROP and unfavorable structural outcomes at week 52 of chronological age. 

Findings from both trials showed that nearly 80% of patients who received aflibercept achieved an absence of active ROP and unfavorable structural outcomes at 52 weeks of age. Neither trial achieved superiority or inferiority of one arm compared with the other arm. In both trials, more than 92% of all patients who were treated with aflibercept received bilateral injections.

“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.”

Following administration of Eylea, there is a risk of reactivation of abnormal angiogenesis and tortuosity. Infants should be monitored closely after injection until retinal vascularization has completed or until the examiner is assured that reactivation of ROP will not occur.

Eylea is also indicated for the treatment of adults with neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy.

References

1. Eylea^® (aflibercept) Injection approved as the first pharmacologic treatment for preterm infants with retinopathy of prematurity (ROP) by the FDA. News release. Regeneron Pharmaceuticals, Inc. February 8, 2023. Accessed February 9, 2023. https://www.globenewswire.com/news-release/2023/02/08/2604539/0/en/EYLEA-aflibercept-Injection-Approved-as-the-First-Pharmacologic-Treatment-for-Preterm-Infants-with-Retinopathy-of-Prematurity-ROP-by-the-FDA.html.
2. Eylea. Package insert. Regeneron Pharmaceuticals, Inc.; 2023. Accessed February 9, 2023. https://www.regeneron.com/downloads/eylea_fpi.pdf.