Abstract | PURPOSE OF REVIEW:
Mipomersen has been approved by the US Food and Drug Administration as an orphan drug for patients with homozygous familial hypercholesterolemia ( HoFH). In contrast, the European Medicines Agency advised negatively on the use of mipomersen. In this review, we discuss the efficacy and safety considerations for this discrepancy. RECENT FINDINGS: On the basis of the results of clinical trials with mipomersen, safety concerns have been raised regarding cardiovascular risk reduction and development of hepatic steatosis. In addition, (long-term) tolerability concerns have been raised predominantly regarding injection site reactions. A pooled analysis of cardiovascular events in phase III trials with mipomersen did not provide evidence for either a positive or negative effect on cardiovascular disease. Although long-term studies with mipomersen are eagerly awaited, hepatic fat content appears to stabilize after 6-12 months notwithstanding continued mipomersen administration. SUMMARY:
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Authors | Barbara Sjouke, Deepak M W Balak, Ulrich Beuers, Vlad Ratziu, Erik S G Stroes |
Journal | Current opinion in lipidology
(Curr Opin Lipidol)
Vol. 24
Issue 4
Pg. 301-6
(Aug 2013)
ISSN: 1473-6535 [Electronic] England |
PMID | 23759796
(Publication Type: Journal Article, Review)
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Chemical References |
- Anticholesteremic Agents
- Cholesterol, LDL
- Oligonucleotides
- mipomersen
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Topics |
- Animals
- Anticholesteremic Agents
(adverse effects, pharmacology, therapeutic use)
- Cholesterol, LDL
(blood)
- Clinical Trials, Phase III as Topic
- Drug Approval
- Europe
- Fatty Liver
(chemically induced)
- Humans
- Hyperlipoproteinemia Type II
(blood, drug therapy)
- Liver
(drug effects, metabolism)
- Meta-Analysis as Topic
- Oligonucleotides
(adverse effects, pharmacology, therapeutic use)
- United States
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