The Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) recommended the approval of the investigational therapy Praluent (alirocumab) Injection for patients with hypercholesterolemia.
The Committee voted 13 to three (with no abstentions) that Regeneron and Sanofi had sufficiently established that the low-density lipoprotein cholesterol (LDL-C, or bad cholesterol) lowering benefit of Praluent exceeds its risks to support approval in one or more patient populations. If approved by the FDA, Praluent is expected to be the first fully human monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) in the U.S. The Marketing Authorization Application for Praluent in the European Union is currently under review by the European Medicines Agency (EMA). The safety and efficacy of Praluent have not been fully evaluated by any regulatory authority.
"We are pleased with the Committee's recommendation to approve Praluent. Our clinical trial program focused on patients with high unmet need in which Praluent delivered significant reductions in LDL-C on top of statins and other lipid-lowering therapies," said Elias Zerhouni, M.D., President, Global R&D, Sanofi. "Our Phase 3 Praluent development program investigated both a 75 mg and 150 mg dose, providing flexible dosing regimens that can be tailored to individual patient cholesterol lowering needs."
The Committee's recommendation was based on Praluent's benefit-risk profile, following review of efficacy and safety data from more than 5,000 patients across 10 pivotal Phase 3 double-blind trials ranging from six months to two years. Clinical data from the ODYSSEY Phase 3 program show consistent, positive results in reducing LDL-C. Common adverse events that were more frequently reported in patients treated with Praluent than the control groups included injection site reaction and pruritus (itching).
"The discovery of PCSK9 as a powerful regulator of cholesterol levels and cardiovascular disease was one of the most important human genetic advances of the last decade," said George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron and President, Regeneron Laboratories. "Today's outcome brings us one step closer to translating this genetics-based discovery into a treatment that may help the many patients in need of additional cholesterol lowering."
The Advisory Committee's recommendation will be considered by the FDA in its review of the Biologics License Application (BLA) for Praluent. The FDA is not bound by the Committee's recommendation, but takes its advice into consideration when reviewing investigational medicines. The BLA for Praluent was accepted for priority review by the FDA with a target action date of July 24, 2015.