FDA Approves Regeneron’s EYLEA® as the First Pharmacologic Treatment for Preterm Infants with ROP

FDA Approves Regeneron’s EYLEA® as the First Pharmacologic Treatment for Preterm Infants with ROP

February 09, 2023

Regeneron Pharmaceuticals announced that the FDA granted its approval for the use of EYLEA® (aflibercept) Injection as a treatment option for preterm infants suffering from retinopathy of prematurity (ROP). This marks the first time that EYLEA has received approval for pediatric use and expands its indications to five different 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, M.D., Ph.D., 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. We thank the investigators and the many families who participated in the clinical trials.”

Annually, between 1,100 to 1,500 infants in the United States are diagnosed with ROP, a rare eye disease, which requires medical intervention. This condition affects preterm infants born before 31 weeks of pregnancy or weighing less than 1,500 grams (3.3 lbs) at birth. As full-term infants have fully developed retinal blood vessels, preterm infants are vulnerable to abnormal retinal blood vessel growth (neovascularization) and the subsequent detachment of the retina, resulting in vision loss or blindness. Mild cases of ROP may improve on their own, but treatment is necessary in some cases to prevent significant visual impairment.

“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.”

About EYLEA®

EYLEA® (aflibercept) Injection 2 mg (0.05 mL) is a prescription medicine approved for the treatment of patients with Wet Age-related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), Diabetic Retinopathy (DR), and Retinopathy of Prematurity (ROP).

The drug functions as a VEGF inhibitor. It is created to prevent the development of new blood vessels in the eye by inhibiting the activity of VEGF-A and PLGF, two growth factors that play a role in ocular angiogenesis. The safety and effectiveness of EYLEA have been established through extensive research, including eight significant Phase 3 trials, over 11 years of real-world use, and more than 57 million EYLEA injections administered worldwide.