FDA grants Breakthrough Therapy designation to EYLEA Injection for treatment of diabetic retinopathy

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has granted EYLEA^® (aflibercept) Injection Breakthrough Therapy designation for the treatment of diabetic retinopathy in patients with diabetic macular edema (DME). The designation is based on positive results in two Phase 3 trials (VIVID-DME and VISTA-DME), in which EYLEA demonstrated a statistically significant improvement in a pre-specified measure of diabetic retinopathy in patients with DME after two years of treatment.

"Millions of people in the U.S. are living with diabetic eye diseases that can cause vision loss and even blindness," said George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron and President of Regeneron Laboratories. "There are no FDA-approved medicines for diabetic retinopathy and we look forward to working closely with the FDA to potentially bring EYLEA to these patients as soon as possible. We plan to submit a supplemental Biologics License Application (sBLA) in the U.S. for diabetic retinoapthy in patients with DME later this year."

In the Phase 3 VIVID-DME trial in diabetic retinopathy patients with DME, 29 percent of evaluable patients in the 2Q4 group (monthly) and 33 percent of evaluable patients in the 2Q8 group (every two months, after 5 initial monthly injections) treated with EYLEA experienced at least a 2-step improvement on the diabetic retinopathy severity scale (DRSS), a grading system measuring the degree of retinopathy, compared to 8 percent of patients in the laser control group (p less than 0.001). In the Phase 3 VISTA-DME trial in diabetic retinopathy patients with DME, 40 percent of evaluable patients in both the 2Q4 and 2Q8 groups treated with EYLEA experienced at least a 2-step improvement on the DRSS compared to 17 percent of patients in the laser control group (p less than 0.0001). The most frequent ocular adverse events (AEs) observed in the VIVID-DME trial were conjunctival hemorrhage, cataract, and increased intraocular pressure. The most frequent ocular AEs observed in the VISTA-DME trial were conjunctival hemorrhage, eye pain, and vitreous floaters. The two-year results from these trials on the primary endpoint of best-corrected visual acuity (BCVA) and overall safety have been previously announced and are available here.

The Breakthrough Therapy designation was created by the FDA to expedite the development and review of drugs for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the fast track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

EYLEA is approved in the United States, European Union (EU) and other countries for the treatment of wet age-related macular degeneration (AMD), macular edema following central retinal vein occlusion (CRVO), and DME. Regulatory submissions have been made for EYLEA in the U.S. and EU for macular edema following branch retinal vein occlusion (BRVO).