Regeneron gets CRL as it shoots for approval of Eylea in diabetic eye disease

Regeneron’s top-selling blockbuster Eylea has shown good results for a new and potentially lucrative indication, hitting its one-year endpoints in a phase 3 trial for treatment of diabetic eye disease. Its prefilled syringe, however, did not.

The FDA has issued a complete response letter (CRL) tied to the manufacturing of the pre-filled syringe and asked for more info about that and the supply processes. In addition, it wants Regeneron to complete a usability study evaluating a single injection of the Eylea pre-filled syringe in approximately 30 patients as part of the pre-approval process.

Regeneron said it expects to complete the study and get the information to the FDA in early 2019 and does not expect the process to delay launch of the Eylea pre-filled syringe. It has a May 13, 2019 target date for potential approval of Eylea in NPDR.

The news came as the Tarrytown, New York-based Regeneron reported Eylea hit the primary endpoint in a phase 3 trial of patients taking Eylea on an every 8- and every 16-week interval for moderately severe and severe diabetic retinopathy. Without treatment, more than one third of patients developed a vision-threatening complication or diabetic macular edema within one year, Regeneron said.

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It said it was the first trial in NPDR to show both a reduction in vision-threatening complications and in development of diabetic macular edema. Moderately severe and severe diabetic retinopathy is the leading cause of vision impairment and blindness among working-age adults.

“Eylea was able to reduce these complications by 68%-85% even with every four-month dosing, and moreover was able to reverse the anatomic severity of the disease,” George D. Yancopoulos, Regeneron's chief scientific officer said in a statement.

This is important because Regeneron's foundational drug is under assault by other treatments. Eylea generated about $3.7 billion in sales for Regeneron last year, with about 70% of its U.S. sales coming from patients with wet age-related macular degeneration (AMD). With new drug candidates threatening Eylea’s lock on that indication, the company has been testing the blockbuster drug in other areas.

Another one of those areas is for the treatment of NPDR in patients without DME. A separate phase 3 trial is evaluating patients in that area.

Last year, Leerink Partners’ Geoffrey Porges said based on what was known so far about Eylea’s use in NPDR, it could turn out to be a significant revenue generator for the company. He suggested that Eylea could “achieve higher market share than it currently holds in its existing wet AMD indication.”