Four years after scoring an approval to treat diabetic retinopathy—a disease that strikes more than a third of those with diabetes and is the primary cause of blindness in the U.S.—Regeneron’s Eylea has gained an FDA nod for the infant version of the disorder.
The regulator has signed off on Eylea as a treatment for retinopathy of prematurity (ROP). The disease often strikes babies who are born less than 31 weeks into pregnancy or weighing less than 3.3 pounds.
Eylea becomes the only pharmacologic answer for ROP. The other alternative is laser surgery, which can destroy retinal tissue and lead to significant long-term complications such as nearsightedness and peripheral vision loss.
“Laser photocoagulation (is) a complex and lengthy procedure that permanently ablates retina tissue and is stressful not only for infant parents but also for the family navigating a delicate time after preterm birth,” Regeneron CEO Len Schleifer said in a release.
Three months ago, Regeneron's collaborator on Eylea, Bayer, gained a positive recommendation in Europe from the Committee for Medicinal Products for Human Use (CHMP) to treat ROP. Japan gave Eylea a thumbs up for ROP in September of last year.
ROP is the fifth eye disease Eylea has been sanctioned to treat. Its first FDA endorsement came for age-related macular degeneration (AMD) in 2011, followed three years later by a nod to treat diabetic macular edema (DME). The VEGF inhibitor became a blockbuster in 2014 and last year generated $6.2 billion in sales in the U.S. Bayer, which collaborates with Regeneron on Eylea outside the U.S., has yet to present its quarterly figures.
Regeneron got the approval with less-than-ideal clinical data. In two phase 3 trials—one sponsored by Regeneron and the other by Bayer—80% of Eylea patients were clear of ROP with no unfavorable structural damage at 52 weeks of age. The trials compared Eylea to laser surgery.
In both trials, however, Eylea didn't meet its endpoint of non-inferiority to laser photocoagulation. Regeneron noted that efficacy in both trials for laser surgery exceeded what was observed in previous studies. In both trials, there was no significant difference between the rate of adverse events between the two groups.
ROP affects between 1,100 and 1,500 babies born each year in the U.S. and occurs because blood vessels aren’t yet fully developed, leading to potential retinal detachment. Mild cases can be reversed through treatment, but others require laser surgery to prevent visual impairment or blindness.
In October—based on data from two trials—the FDA granted Eylea a six-month pediatric exclusivity extension. Instead of losing patent protection on the treatment in the U.S. in November of this year, it will be delayed to May of 2024.
It’s not certain when Eylea would face biosimilar competition. In the meantime, it is feeling heat from Roche’s Vabysmo, which was approved simultaneously for AMD and DME a year ago, and has more staying power for many patients, requiring three shots per year as opposed to six for Eylea. Both treatments are injected into the eyeball.
Last year, Regeneron answered with impressive data on its longer-acting version of Eylea, which sets the company up for possible approval later this year.