title: Efficacy and safety of mipomersen sodium (Kynamro).
abstract: BACKGROUND
  Mipomersen is a first-in-class drug indicated as an adjunct to lipid-lowering medications and diet to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB), total cholesterol (TC) and non-high density lipoprotein-cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
  
  
  METHODS
  This article summarizes the efficacy and safety profile of mipomersen based on literature, public materials available from the Endocrinologic and Metabolic Drugs Advisory Committee meeting (FDA) in review of the New Drug Application (NDA 203568) and the recent product label.
  
  
  CONCLUSIONS
  Patients suffering from HoFH are characterized by elevated levels of LDL-C and are, therefore, at severely increased risk for cardiovascular disease (CVD). Currently available lipid-lowering therapies (LLT), such as statins, have been shown to lower LDL-C levels and CVD risk. However, in patients suffering from HoFH, additional therapy is urgently needed to further decrease LDL-C levels and CVD risk. Mipomersen (Kynamro) has recently been approved by the FDA as a novel LLT modality in patients with HoFH. Mipomersen has been show to result in highly relevant absolute LDL-C reductions in HoFH patients, and given the undisputed causal relationship between LDL-C levels and CVD risk, this additional LDL-C lowering is expected to result in a robust CVD risk reduction.
authors.affiliation: Academic Medical Center, Department of Vascular Medicine, Meibergdreef 9, Amsterdam, 1105 AZ, Netherlands.
authors.forename: Kees
authors.initials: K
authors.lastname: Hovingh
authors.forename: Joost
authors.initials: J
authors.lastname: Besseling
authors.forename: John
authors.initials: J
authors.lastname: Kastelein
mesh_terms: D000818:Animals; D000924:Anticholesteremic Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy*; D009841:Oligonucleotides / Q000009:adverse effects* / Q000627:therapeutic use*
chemical_list: D000924:Anticholesteremic Agents; D009841:Oligonucleotides; C524142:mipomersen