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Updated: Apr 21, 2026

Differential Effects of Lipid-lowering Drugs in Modulating Morphology of Cholesterol Particles
Published on: November 10, 2017
[New lipid lowering agents]
Insights
New therapies effectively lower LDL-cholesterol in severe hypercholesterolemia cases where statins are insufficient. These novel agents show promise, but long-term studies are needed to confirm their safety and cardiovascular benefits.
Area of Science:
- Cardiovascular Medicine
- Pharmacology
- Genetics
Background:
- Statins are primary treatments for hypercholesterolemia, reducing cardiovascular risks.
- Severe hypercholesterolemia (e.g., familial hypercholesterolemia) or statin intolerance can limit statin efficacy.
- Novel lipid-lowering agents offer alternative treatment strategies.
Purpose of the Study:
- To review the effectiveness and safety of new lipid-lowering agents for severe hypercholesterolemia.
- To assess these agents in patients with homozygous or heterozygous familial hypercholesterolemia.
- To evaluate their role in statin-intolerant populations.
Main Methods:
- Review of studies on novel lipid-lowering therapies.
- Inclusion of agents like Lomitapide, Mipomersen, and PCSK9 inhibitors.
- Analysis of short-term safety and efficacy data.
Main Results:
- New therapies demonstrate significant LDL-cholesterol reduction in severe hypercholesterolemia.
- Short-term studies indicate an acceptable safety profile, particularly in homozygous familial hypercholesterolemia.
- Some agents show potential for statin-intolerant patients.
Conclusions:
- Novel agents are effective for managing severe hypercholesterolemia when statins are insufficient.
- Further long-term studies are essential to fully ascertain the safety, efficacy, and cardiovascular impact of these new therapies.
Abstract:
Statins are the preferred treatment for hypercholesterolemia and several studies have demonstrated their long-term safety and efficacy in reducing cardiovascular morbidity and mortality. However, in some cases of severe hypercholesterolemia such as homozygous and heterozygous familial hypercholesterolemia or statin intolerant patients, statins can be less efficient. In recent years, new lipid-lowering agents with novel mechanisms of action have been developed to reduce LDL-cholesterol in patients with severe hypercholesterolemia, associated or not to conventional lipid-lowering therapy. These therapies include microsomal transfer protein inhibitor (Lomitapide), antisense oligonucleotide to Apo B100 (Mipomersen) and monoclonal antibodies against Proprotein convertase subtilisin/kexin type 9 (PCSK9). Different studies have shown the great effectiveness of these new therapies. Short-term studies confirmed their adequate security profile, especially in patients with homozygous familiar hypercholesterolemia or severe hypercholesterolemia. Some of these agents have been also tested in statin-intolerant patients. However, long-term studies are needed to evaluate their safety, effectiveness and impact on cardiovascular risk reduction.
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