FDA Approves Praluent for High LDL Cholesterol Patients; European Agency Recommends Approval

July 24, 2015—Regeneron Pharmaceuticals, Inc. and Sanofi announced that the US Food and Drug Administration (FDA) approved Praluent (alirocumab) injection, which is a PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor. In the United States, Praluent is indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein (LDL) cholesterol. The effect of Praluent on cardiovascular morbidity and mortality has not been determined, advised the companies.

Beginning next week, Praluent will be available in the United States in two doses (75 mg and 150 mg). Both doses of Praluent are available in a single 1-mL injection delivered in a single-dose prefilled pen or syringe that patients self-administer every 2 weeks.

The companies also announced that the European Medicine Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion for the marketing authorization of Praluent, recommending approval for both the 75-mg and 150-mg dose for use in certain adult patients with hypercholesterolemia. The European Commission is expected to make a final decision on the Marketing Authorization Application for Praluent in the European Union in late September.

According to the companies, the FDA approval of Praluent was based on data from the pivotal phase 3 ODYSSEY program, which showed consistent, positive results compared to placebo, which included current standard-of-care therapy (statins). In the ODYSSEY LONG TERM trial that evaluated Praluent 150 mg every 2 weeks, Praluent reduced LDL cholesterol by 58% versus placebo at week 24 when added to current standard of care, including maximally tolerated statins. In ODYSSEY COMBO I, Praluent 75 mg every 2 weeks as an adjunct to statins reduced LDL cholesterol by an additional 46% compared to placebo at week 12. At week 24 in the same trial, Praluent reduced LDL cholesterol by 43% compared to placebo. In this study, if additional LDL cholesterol lowering was required based on prespecified criteria at week 8, Praluent was up-titrated to 150 mg at week 12 for the remainder of the trial. Eighty-three percent of patients remained on their initial 75 mg dose, advised the companies.

Christopher Cannon, MD, is a member of the Steering Committee for the phase 3 ODYSSEY clinical trial program. He is Professor of Medicine at Harvard Medical School, Cardiovascular Division at Brigham and Women's Hospital in Boston, Massachusetts.

In the companies’ press release, Dr. Cannon commented, “For patients with high LDL, or bad, cholesterol the primary focus of treatment is to lower their levels, but many patients today do not achieve recommended levels despite lifestyle modifications and treatment with statins. In the ODYSSEY clinical trial program, two doses of alirocumab showed significant LDL cholesterol lowering in a variety of patients who were not able to adequately lower their LDL cholesterol with current standard of care alone. The majority of patients achieved their LDL-lowering goals with the 75-mg dose, when added to maximally tolerated dose of a statin, with a generally acceptable safety profile.”