Regeneron's BLA for fusion protein

Tarrytown, NY-Regeneron Pharmaceuticals may know within 6 to 10 months the results of the FDA’s review of the company’s biologics license application (BLA) related to a fusion protein (VEGF Trap-Eye) for the treatment of neovascular age-related macular degeneration (AMD). If the review is favorable, use of the fusion protein ultimately could mean fewer injections per year for patients being treated for the disease.

Under the Prescription Drug User Fee Act (PDUFA), the standard review time for a BLA from submission to FDA action is 10 months. The company’s submission included a request for priority review, which, if granted, would shorten the FDA’s targeted goal for review time to 6 months.

“There have been significant advances in the treatment of wet AMD in recent years. However, the need for monthly intravitreal injections to obtain optimal vision gains has resulted in a significant burden for physicians, patients, and their caregivers,” said Leonard S. Schleifer, MD, PhD, president and chief executive officer of Regeneron. “We are extremely proud to have conducted the largest global phase III clinical program in patients with wet AMD, which demonstrated that patients treated with [2 mg of the fusion protein] every 2 months, following three loading doses, were able to be dosed with fewer injections over 1 year without compromising efficacy. We look forward to working closely with the FDA to bring this potentially important new medicine to patients with wet AMD.”

Submission of the BLA is based on the positive results from two phase III trials, the North American VIEW 1 trial and the global VIEW 2 trial. In these trials, all regimens of the fusion protein, including dosing at 2 mg every 2 months following three loading doses, successfully met the primary endpoint of non-inferiority compared with the current standard of care, ranibizumab 0.5 mg (Lucentis, Genentech) dosed every month.

The primary endpoint analysis was statistical non-inferiority in the proportion of patients whose vision was maintained or improved over 52 weeks compared with patients treated with ranibizumab. A generally favorable safety profile was observed for both the fusion protein and ranibizumab. The ocular adverse events were balanced across all treatment groups in both studies. No notable differences in non-ocular adverse events existed between the study arms.