FDA panel to review PCSK9 inhibitor

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee is scheduled to vote June 9 on the safety and effectiveness of the monoclonal antibody alirocumab as a treatment for patients with hypercholesterolemia.

Alirocumab (Praluent, Regeneron Pharmaceuticals and Sanofi Aventis) is a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor that is designed to reduce low-density lipoprotein (LDL) cholesterol and other atherogenic lipoproteins in patients with non-familial and heterozygous familial hypercholesterolemia. The treatment, which is injected, can be administered either in combination with a statin or as monotherapy in statin-intolerant patients.

The FDA granted Regeneron Pharmaceuticals and Sanofi Aventis priority review early this year and announced that it would make its decision no later than July 24.

Results from Phase 3 clinical trials have been positive. ODYSSEY ALTERNATIVE, an international, randomized, double-blind, double-dummy placebo-controlled study, found that at 24 weeks, patients in the alirocumab group had a 45 percent reduction in LDL cholesterol from baseline compared with a 14.6 percent reduction in patients treated with ezetimibe. In addition, 42 percent for the alirocumab group vs. 4 percent for the ezetimibe group reached target lipid levels.

Patients treated with alirocumab also reported fewer skeletal-muscle-related treatment-emergent adverse events and fewer discontinued treatment as a result.

The panel will discuss safety issues, including potential adverse outcomes such as diabetes or neurological and neurocognitive events, and the benefits and risks. The same panel will review another cholesterol-lowering treatment, evolocumab (Repatha, Amgen), on June 10.