The US Food and Drug Administration (FDA) has approved aflibercept (Eylea) to treat preterm infants with retinopathy of prematurity. This the fifth indication approved for aflibercept but the first approved orphan drug indication.

ROP is caused by abnormal development of retinal blood vessels in premature infants. Babies weighing less than 1250-1500 grams (3 – 3.3 lb) and born before 31 weeks of gestation are at highest risk. Approximately 14,000 premature babies have ROP per year but most of those cases are mild. In about 1,100 to 1,500 of the cases, the condition is severe enough to require medical treatment.

The FDA approval of aflibercept was largely based on data from  two randomized global Phase 3 trials – FIREFLEYE (N=113) and BUTTERFLEYE (N=120) – investigating aflibercept 0.4 mg versus laser surgery in infants with ROP. In both trials, approximately 80% of aflibercept -treated infants achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age.  Data from the FIREFLEYE trial has been published in JAMA in July 2022.

No new adverse events concerns were observed in either trial.

Laser photocoagulation has been the standard treatment of ROP but this procedure is associated with irreversible visual field loss and a high level of myopia. Upregulation of vascular endothelial growth factor (VEGF) is observed in ROP and aflibercept is an anti-VEGF agent.

Aflibercept is also approved to treat numerous other eye conditions, including wet age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy.

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