Approval Recommended for Regeneron and Sanofi's Praluent Injection to Treat Hypercholesterolemia

June 9, 2015—Regeneron Pharmaceuticals, Inc. and Sanofi announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the US Food and Drug Administration (FDA) recommended approval of the investigational Praluent (alirocumab) injection. Praluent is a fully human monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9).

The committee voted 13 to three (with no abstentions) that Regeneron and Sanofi had sufficiently established that the low-density lipoprotein cholesterol (LDL-C) lowering benefit of Praluent exceeds its risks to support approval in one or more patient populations. The FDA accepted the Biologics License Application for Praluent for priority review, with a target action date of July 24, 2015.

According to the company’s press release, the committee’s recommendation was based on Praluent’s benefit-risk profile and following review of efficacy and safety data from more than 5,000 patients across 10 pivotal phase 3, double-blind trials ranging from 6 months to 2 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.

In the announcement, Sanofi advised that the clinical trial program focused on patients with a high unmet need in which Praluent delivered significant reductions in LDL-C in addition to statins and other lipid-lowering therapies. The phase 3 Praluent development program investigated both a 75- and 150-mg dose to provide flexible dosing regimens that can be tailored to individual patient cholesterol-lowering needs.

Regeneron noted that 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. The EMDAC committee recommendation advances the process of translating this genetics-based discovery into a treatment that may help many patients who are in need of additional cholesterol lowering.

The companies advised that the EMDAC committee’s recommendation will be considered by the FDA in its review of the Biologics License Application for Praluent. The FDA is not bound by the committee’s recommendation, but takes its advice into consideration when reviewing investigational medicines.

The Marketing Authorization Application for Praluent in the European Union is currently under review by the European Medicines Agency. The safety and efficacy of Praluent have not been fully evaluated by any regulatory authority, stated the companies.