Regeneron Pharmaceuticals, Inc. (and Bayer) has received FDA approval for Eylea (aflibercept) injection for the treatment of retinopathy of prematurity (ROP) in preterm infants.
Refresh me on Eylea.
Eylea is a vascular endothelial growth fact (VEGF) inhibitor that’s administered as an intravitreal injection It inhibits new blood vessel growth and reduces vascular permeability in the eye by blocking major growth factors involved in ocular angiogenesis (VEGF-A and placental growth factor).
How does it work?
The formulation is designed to be injected into the eye 6-8 times a year. For infants with ROP, treatment will require extended periods of monitoring.
Give me some background.
The FDA granted Eylea orphan drug designation in 2019 and is currently already approved for wet age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, diabetic retinopathy, and now ROP..
What did clinical trials show?
Two randomized global phase 3 clinical trials assessed 0.4 mg of Eylea compared to laser photocoagulation in infants with ROP.
Both trials—FIREFLEYE (NCT04004208) and BUTTERFLEYE (NCT04101721) reported an estimated 80% of infant patients treated with Elyea were without active ROP as well as unfavorable structural outcomes by age 52 weeks.
How about adverse reactions?
Ocular adverse events (AEs) were observed in 39% of Eylea patients versus 37% in laser in FIREFLEYE and 18% versus 26% in BUTTERFLEYE serious AEs were observed in 8% for both Eylea and laser groups in FIREFLEYE and 6.5% vs 11%, respectively, in BUTTERFLEYE.
In reference to side effects, most commonly reported among preterm infants were retinal detachment along with conjunctival hyperemia and elevated intraocular pressure.
What else should I know?
In October 2022, the FDA granted Regeneron pediatric exclusivity for Elyea, extending its U.S. market exclusivity to May 17, 2024—an additional 6 months beyond its original date—as a result of these additional phase 3 programs.
Significance?
This approval marks the first pharmacological treatment for ROP in infants as well as the fifth approved indication for Eylea to treat retinal conditions caused by ocular angiogenesis.