Regeneron Pharmaceuticals, Inc. Wins Breakthrough Therapy Designation From FDA For EYLEA

September 16, 2014

By Krystle Vermes, BioSpace.com Breaking News Staff

Regeneron Pharmaceuticals, Inc. has announced today that the U.S. Food and Drug Administration has granted the its diabetic drug EYLEA a breakthrough therapy designation for the treatment of diabetic retinopathy in patients with diabetic macular edema.

This news comes after data of two Phase III trials of EYLEA that was overwhelmingly positive.

EYLEA, which is also known as aflibercept, showed a significant improvement in a pre-specified measure of diabetic retinopathy in patients with DME after two years of treatment.

Diabetic retinopathy is a complication that results from diabetes, which causes damage to the retina. It may lead to poor vision or vision loss over time. Approximately 29 million Americans adults are currently living with diabetes.

"Millions of people in the U.S. are living with diabetic eye diseases that can cause vision loss and even blindness," said George Yancopoulos, 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.”

The company plans to submit a supplemental Biologics License Application (sBLA) in the U.S. for diabetic retinoapthy in patients with DME later this year.

Trial Design and Results
In the Phase III VIVID-DME trial, 29 percent of participants who received EYLEA once per month and 33 percent of patients who received it every two months (after five initial monthly injections) showed a 2-step improvement on the diabetic retinopathy severity scale. This scale, which measures the degree of retinopathy in patients, was developed to predict the risk of progression from non-proliferative diabetic retinopathy to proliferative diabetic retinopathy.

The most common side effects of EYLEA in participants were conjunctival hemorrhage, eye pain and vitreous floaters.

The FDA created the Breakthrough Therapy designation to speed up the development and review of drugs designed for life-threatening conditions. Drugs that receive this designation must show evidence of a substantial improvement on a clinically significant endpoint over available therapies, or a placebo.

EYLEA is currently approved in the U.S., European Union and several other countries for the treatment of wet age-related macular degeneration, macular edema following central retinal vein occlusion and DME.