Mipomersen (Kynamro): A novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia

P and T

Volumn 39, Issue 2, 2014, Pages 119-122

  Wong, Elaine ^a   Goldberg, Tamara ^a  

^a LONG ISLAND UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; APOLIPOPROTEIN B100; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; EZETIMIBE; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; HYPOCHOLESTEROLEMIC AGENT; ISOTRETINOIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MICROSOMAL TRIGLYCERIDE TRANSFER PROTEIN INHIBITOR; MIPOMERSEN; PARACETAMOL; PCSK9 INHIBITOR; PLACEBO; SIMVASTATIN; SQUALENE SYNTHASE INHIBITOR; TAMOXIFEN; TAMOXIFEN CITRATE; THYROID HORMONE DERIVATIVE; UNCLASSIFIED DRUG; VERY LOW DENSITY LIPOPROTEIN CHOLESTEROL;

CARDIOVASCULAR RISK; DRUG COST; DRUG MECHANISM; DRUG SAFETY; FAMILIAL HYPERCHOLESTEROLEMIA; HUMAN; ISCHEMIC HEART DISEASE; MULTICENTER STUDY (TOPIC); NOTE; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); RISK ASSESSMENT;

EID: 84894047761     PISSN: 10521372     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Note

References (21)

1. Goldberg AC, Hopkins PN, Toth PP, et al. Familial hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients. Clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 2011;5:133-140.
2. Hovingh GK, Davidson MH, Kastelein JJ, O'Connor AM. Diagnosis and treatment of familial hypercholesterolaemia. Eur Heart J 2013;34(13):962-971.
3. Ito MK, McGowan MP, Moriarty PM. Management of familial hypercholesterolemias in adult patients: Recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 2011;5:S38-S45.
4. Radar DJ, Hobbs HH. Disorders of lipoprotein metabolism. In: Longo DL, Fauci AS, Kasper DL, et al., eds. Harrison's Principles of Internal Medicine, 18th ed. New York: McGraw-Hill; 2012:3145-3161.
5. Third Report of the National Cholesterol Education Program (NCEP) Expert Panel on Detection, Evaluation, and Treatment of High Blood Cholesterol in Adults (Adult Treatment Panel III), final report. Circulation 2002;106(25):3143-3421.
6. Robinson JG. Management of familial hypercholesterolemia: A review of the recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Manag Care Pharm 2013;19(2):139-149.
7. Goldberg AC. Novel therapies and new targets of treatment for familial hypercholesterolemia. J Clin Lipidol 2010;4(5):350-356.
8. Kynamro (mipomersen sodium) injection solution for subcutaneous injection, prescribing information. Cambridge, Mass.: Genzyme Corp.; 2013. Available at: www.kynamro.com/~/media/Kynamro/ Files/kynamro-pi.pdf. Accessed March 23, 2013.
9. FDA News Release: FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder, January 29, 2013. Available at: www.fda.gov/NewsEvents/ Newsroom/PressAnnouncements/ ucm337195.htm. Accessed April 2, 2013.
10. FDA, Regulatory Information: Orphan Drug Act, updated July 18, 2013. Available at: www.fda.gov/RegulatoryInformation/ Legislation/FederalFoodDrugandCosmeticActFDCAct/ SignificantAmendmentstotheFDCAct/ OrphanDrugAct/ default.htm. Accessed April 2, 2013.
11. Davis RA. Cell and molecular biology of the assembly and secretion of apolipoprotein B-containing lipoproteins by the liver. Biochim Biophys Acta 1999;1440:1-31.
12. Dias N, Stein CA. Antisense oligonucleotides: Basic concepts and mechanisms. Mol Cancer Ther 2002;1:347-355.
13. Ricotta DN, Frishman W. Mipomersen: A safe and effective antisense therapy adjunct to statins in patients with hypercholesterolemia. Cardiol Rev 2012;20:90-95.
14. Thomas T, Ginsberg H. Development of apolipoprotein B antisense molecules as a therapy for hyperlipidemia. Curr Atheroscler Rep 2010;12(1):58-65.
15. 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(16):1729-1735.
16. Akdim F, Tribble DL, Flaim JD. Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia. Eur Heart J 2011;32:2650-2659.
17. 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 hypercholesterolemia: A randomised, double-blind, placebocontrolled trial. Lancet 2010;375:998-1006.
18. McGowan MP, Tardif J-C, Ceska R, et al. Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy. PLoS One, November 13, 2012 (online).
19. 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.
20. Yu RZ, Geary RS, Flaim JD, et al. Lack of pharmacokinetic interaction of mipomersen sodium (ISIS 301012), a 2́-O-methoxyethyl modified antisense oligonucleotide targeting apolipoprotein B-100 messenger RNA, with simvastatin and ezetimibe. Clin Pharmacokinet 2009;48:39-50.
21. Red Book Online. Ann Arbor, Mich.: Truven Health Analytics. Accessed April 4, 2013.