[Image courtesy of Regeneron/Bayer]

Regeneron Pharmaceuticals (Nasdaq:REGN) has announced that the FDA has granted Priority Review of the supplemental Biologics License Application (sBLA) for Eylea (aflibercept) Injection to treat retinopathy of prematurity (ROP) in preterm infants.

First approved in 2011 to treat wet age-related macular degeneration, aflibercept is a blockbuster drug. Last year, it generated $9.2 billion for Regeneron and aflibercept co-developer Bayer. The drug is also indicated for diabetic retinopathy, diabetic macular edema and macular edema following retinal vein occlusion. 

The Tarrytown, New York–based company has conducted two randomized Phase 3 studies testing aflibercept against laser in infants with retinopathy of prematurity. Both the FIREFLEYE and BUTTERFLEYE studies tested a 0.4 mg dose of aflibercept. Both studies found that roughly 80% of aflibercept recipients had an absence of ROP and unfavorable structural outcomes at 52 weeks of age.  

Regeneron acknowledges that non-inferiority was not met in the study at week 24 as laser showed comparable levels of efficacy. The company notes, however, that aflibercept was associated with a substantial reduction in time needed to complete treatment administration in patients. 

A common cause of childhood blindness, ROP is more common in infants who weigh less than 1,500 grams (3.3 lbs) pounds at birth. The disease tends to affect infants born before 31 weeks of pregnancy. 

There are five distinct stages involved in ROP. In the fifth stage, the retina detaches completely, potentially causing vision loss or blindness.

In the U.S., between 1,100 to 1,500 infants develop ROP necessitating medical treatment.

Earlier this year, JAMA published data from the FIREFLEYE study

Regeneron presented data from the BUTTERFLEYE study at the ROP Update 2022 meeting. 

The company anticipates an FDA response to its sBLA by early 2023. 

FDA granted orphan drug designation for aflibercept in ROP in 2019. 

Regeneron has exclusive rights to aflibercept in the U.S., while Bayer maintains exclusive rights in the rest of the world. 

Viatris (Nasdaq:VTRS) and J&J unit Momenta Pharmaceuticals (NYSE:JNJ) are working on developing an aflibercept biosimilar known as MYL-1701P. The companies recently presented data at the American Academy of Ophthalmology (AAO) 2022 Annual Meeting in Chicago, showing that the MYL-1701P had similar rates of meaningful visual acuity (VA) change in patients with diabetic macular edema compared with patients who received aflibercept.

In related news, Regeneron recently announced that a 8 mg dose of aflibercept was associated with sustained treatment intervals in patients with neovascular eye disease in two late-stage trials.