Is mipomersen ready for clinical implementation? A transatlantic dilemma

Current Opinion in Lipidology

Volumn 24, Issue 4, 2013, Pages 301-306

  Sjouke, Barbara ^a   Balak, Deepak M W ^b   Beuers, Ulrich ^a   Ratziu, Vlad ^c,d   Stroes, Erik S G ^a  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

^b ERASMUS MEDICAL CENTER   (Netherlands)

^c PITIÉ SALPÊTRIÈRE HOSPITAL   (France)

^d SORBONNE UNIVERSITÉ   (France)

Author keywords

Cardiovascular disease; Homozygous familial hypercholesterolemia; Mipomersen; Steatosis

Indexed keywords

ALANINE AMINOTRANSFERASE; ANTIHISTAMINIC AGENT; BETA INTERFERON; CORTICOSTEROID; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; INTERLEUKIN 1 DERIVATIVE; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MIPOMERSEN; PLACEBO; RECOMBINANT TUMOR NECROSIS FACTOR ALPHA;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; CARDIOMETABOLIC RISK; CARDIOVASCULAR DISEASE; CARDIOVASCULAR RISK; CEREBROVASCULAR ACCIDENT; DRUG EFFICACY; DRUG MECHANISM; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; FAMILIAL HYPERCHOLESTEROLEMIA; FAT CONTENT; FATTY LIVER; FLU LIKE SYNDROME; FOLLOW UP; HEART INFARCTION; HEMATOMA; HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA; HUMAN; INFLAMMATION; INJECTION SITE ERYTHEMA; INJECTION SITE PAIN; INJECTION SITE REACTION; LIPID STORAGE; LIVER DISEASE; MULTIPLE SCLEROSIS; NONHUMAN; PATIENT COMPLIANCE; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; RHEUMATOID ARTHRITIS; RISK REDUCTION; SIDE EFFECT; SKIN DISCOLORATION;

ANIMALS; ANTICHOLESTEREMIC AGENTS; CHOLESTEROL, LDL; CLINICAL TRIALS, PHASE III AS TOPIC; DRUG APPROVAL; EUROPE; FATTY LIVER; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; LIVER; META-ANALYSIS AS TOPIC; OLIGONUCLEOTIDES; UNITED STATES;

EID: 84880587999     PISSN: 09579672     EISSN: 14736535     Source Type: Journal    
DOI: 10.1097/MOL.0b013e328362dfd9     Document Type: Review

References (46)

1. Goldstein JL, Hobbs HH, Brown MS. Familial hypercholesterolemia. In: Scriver C, Beaudet A, Sly W, Valle D, editors. The metabolic and molecular bases of inherited disease, 8th ed. New York: McGraw-Hill; 2001. pp. 2863-2913.
2. Pijlman AH, Huijgen R, Verhagen SN, et al. Evaluation of cholesterol lowering treatment of patients with familial hypercholesterolemia: a large crosssectional study in the Netherlands. Atherosclerosis 2010; 209:189-194.
3. Catapano AL, Reiner Z, De Backer G, et al. ESC/EAS Guidelines for the management of dyslipidaemias. The Task Force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). Atherosclerosis 2011; 217:3-46.
4. Wierzbicki AS, Humphries SE, Minhas R. Familial hypercholesterolaemia: summary of NICE guidance. BMJ 2008; 337:a1095.
5. Raal FJ, Pilcher GJ, Panz VR, et al. Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy. Circulation 2011; 124:2202-2207.
6. Szczepiorkowski ZM, Winters JL, Bandarenko N, et al. Guidelines on the use of therapeutic apheresis in clinical practice - evidence-based approach from the Apheresis Applications Committee of the American Society for Apheresis. J Clin Apher 2010; 25:83-177.
7. Thompson GR. Recommendations for the use of LDL apheresis. Atherosclerosis 2008; 198:247-255.
8. Thompson GR, Barbir M, Davies D, et al. Efficacy criteria and cholesterol targets for LDL apheresis. Atherosclerosis 2010; 208:317-321.
9. Mullick AE, Fu W, Graham MJ, et al. Antisense oligonucleotide reduction of apoB-ameliorated atherosclerosis in LDL receptor-deficient mice. J Lipid Res 2011; 52:885-896.
10. Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 2010; 50:259-293.
11. Akdim F, Stroes ES, Sijbrands EJ, et al. Efficacy and safety of mipomersen, an antisense inhibitor of apolipoprotein B, in hypercholesterolemic subjects receiving stable statin therapy. J Am Coll Cardiol 2010; 55:1611-1618.
12. Kastelein JJ, Wedel MK, Baker BF, et al. Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 2006; 114:1729-1735.
13. Akdim F, Tribble DL, Flaim JD, et al. Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia. Eur Heart J 2011; 32:2650-2659.
14. Visser ME, Wagener G, Baker BF, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial. Eur Heart J 2012; 33:1142-1149.
15. Raal FJ, Santos RD, Blom DJ, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet 2010; 375:998-1006.
16. McGowan MP, Tardif JC, Ceska R, et al. Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy. PLoS One 2012; 7:e49006.
17. Gurdasani D, Sjouke B, Tsimikas S, et al. Lipoprotein(a) and risk of coronary, cerebrovascular, and peripheral artery disease: the EPIC-Norfolk prospective population study. Arterioscler Thromb Vasc Biol 2012; 32:3058-3065.
18. Stein EA, Dufour R, Gagne C, et al. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation 2012; 126:2283-2292.
19. FDA briefing report. Available at http://www.fda.gov/downloads/Advisory- Committees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolic DrugsAdvisoryCommittee/UCM323927.pdf.
20. Akdim F, Visser ME, Tribble DL, et al. Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia. Am J Cardiol 2010; 105:1413-1419.
21. Cromwell WC, Thomas GS, Boltje I, et al. Safety and efficacy of mipomersen administered as add-on therapy in patients with hypercholesterolemia and high cardiovascular risk. J Am Coll Cardiol 2011; 57:e504-e505.
22. Neefjes LA, Ten Kate GJ, Alexia R, et al. Accelerated subclinical coronary atherosclerosis in patients with familial hypercholesterolemia. Atherosclerosis 2011; 219:721-727.
23. Smilde TJ, van Wissen S, Wollersheim H, et al. Effect of aggressive versus conventional lipid lowering on atherosclerosis progression in familial hypercholesterolaemia (ASAP): a prospective, randomised, double-blind trial. Lancet 2001; 357:577-581.
24. Baigent C, Blackwell L, Emberson J, et al. Efficacy and safety of more intensive lowering of LDL cholesterol: a meta-analysis of data from 170,000 participants in 26 randomised trials. Lancet 2010; 376:1670-1681.
25. Brunt EM. Pathology of nonalcoholic fatty liver disease. Nat Rev Gastroenterol Hepatol 2010; 7:195-203.
26. Pascale A, Pais R, Ratziu V. An overview of nonalcoholic steatohepatitis: past, present and future directions. J Gastrointest Liver Dis 2010; 19:415-423.
27. Neuschwander-Tetri BA. Hepatic lipotoxicity and the pathogenesis of nonalcoholic steatohepatitis: the central role of nontriglyceride fatty acid metabolites. Hepatology 2010; 52:774-788.
28. Listenberger LL, Han X, Lewis SE, et al. Triglyceride accumulation protects against fatty acid-induced lipotoxicity. Proc Natl Acad Sci USA 2003; 100:3077-3082.
29. Yamaguchi K, Yang L, McCall S, et al. Inhibiting triglyceride synthesis improves hepatic steatosis but exacerbates liver damage and fibrosis in obese mice with nonalcoholic steatohepatitis. Hepatology 2007; 45:1366-1374.
30. Liao W, Hui TY, Young SG, Davis RA. Blocking microsomal triglyceride transfer protein interferes with apoB secretion without causing retention or stress in the ER. J Lipid Res 2003; 44:978-985.
31. Tanoli T, Yue P, Yablonskiy D, Schonfeld G. Fatty liver in familial hypobetalipoproteinemia: roles of the APOB defects, intra-abdominal adipose tissue, and insulin sensitivity. J Lipid Res 2004; 45:941-947.
32. Della CC, Fintini D, Giordano U, et al. Fatty liver and insulin resistance in children with hypobetalipoproteinemia: the importance of aetiology. Clin Endocrinol (Oxf) 2012. [Epub ahead of print]. doi:10.1111/j.1365-2265.2012.04498.x.
33. Visser ME, Lammers NM, Nederveen AJ, et al. Hepatic steatosis does not cause insulin resistance in people with familial hypobetalipoproteinaemia. Diabetologia 2011; 54:2113-2121.
34. Sankatsing RR, Fouchier SW, de Haan HS, et al. Hepatic and cardiovascular consequences of familial hypobetalipoproteinemia. Arterioscler Thromb Vasc Biol 2005; 25:1979-1984.
35. Visser ME, Akdim F, Tribble DL, et al. Effect of apolipoprotein-B synthesis inhibition on liver triglyceride content in patients with familial hypercholesterolemia. J Lipid Res 2010; 51:1057-1062.
36. Crooke RM, Graham MJ, Lemonidis KM, et al. An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. J Lipid Res 2005; 46:872-884.
37. Crooke ST, Geary RS. Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B. Br J Clin Pharmacol 2012. [Epub ahead of print]. doi: 10.1111/j.1365-2125. 2012.04469.x.
38. Callender VD, St Surin-Lord S, Davis EC, Maclin M. Postinflammatory hyperpigmentation: etiologic and therapeutic considerations. Am J Clin Dermatol 2011; 12:87-99.
39. Desmet CJ, Ishii KJ. Nucleic acid sensing at the interface between innate and adaptive immunity in vaccination. Nat Rev Immunol 2012; 12:479-491.
40. Zeltser R, Valle L, Tanck C, et al. Clinical, histological, and immunophenotypic characteristics of injection site reactions associated with etanercept: a recombinant tumor necrosis factor alpha receptor: Fc fusion protein. Arch Dermatol 2001; 137:893-899.
41. Crook S, editor. Antisense drug technology. Principles, strategies and applications, 2nd ed. Boca Raton, Florida: CRC Press; 2008.
42. Balak DM, Hengstman GJ, Cakmak A, Thio HB. Cutaneous adverse events associated with disease-modifying treatment in multiple sclerosis: a systematic review. Mult Scler 2012; 18:1705-1717.
43. Kaiser C, Knight A, Nordstrom D, et al. Injection-site reactions upon Kineret (anakinra) administration: experiences and explanations. Rheumatol Int 2012; 32:295-299.
44. Kerensky TA, Gottlieb AB, Yaniv S, Au SC. Etanercept: efficacy and safety for approved indications. Expert Opin Drug Saf 2012; 11:121-139.
45. Devonshire V, Lapierre Y, Macdonell R, et al. The Global Adherence Project (GAP): a multicenter observational study on adherence to disease-modifying therapies in patients with relapsing-remitting multiple sclerosis. Eur J Neurol 2011; 18:69-77.
46. Van Werven JR, Hoogduin JM, Nederveen AJ, et al. Reproducibility of 3.0 Tesla magnetic resonance spectroscopy for measuring hepatic fat content. J Magn Reson Imaging 2009; 30:444-448.