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Is Lomitapide Safe in Patients with Homozygous Familial Hypercholesterolaemia?


Clinical study deems lomitapide safe for long-term use in Homozygous Familial Hypercholesterolaemia patients

A clinical trial investigates the long-term safety of a drug called lomitapide used for the treatment of homozygous familial hypercholesterolemia. 

Homozygous Familial Hypercholesterolemia (HoFH) is a genetic condition that affects the way the body processes cholesterol, leading to extremely high levels of LDL ("bad") cholesterol in the blood. This puts individuals with HoFH at a high risk of developing heart disease at an early age.

Lomitapide is a medication that works by reducing the amount of LDL cholesterol produced by the liver. However, lomitapide has the potential to lead to idiosyncratic hepatotoxicity. An idiosyncratic hepatotoxicity is an unpredictable form of drug-induced liver injury. It can cause fat build-up in the liver and result in elevated liver enzyme levels.
Hence, due to its potential side effects, it is essential to determine the safety profile of lomitapide for long-term use.

Clinical Study

This clinical study aimed to determine the long-term hepatic safety of lomitapide when used in homozygous familial hypercholesterolemia.

The study was conducted to assess the long-term safety of lomitapide in the liver, which is the organ responsible for metabolizing the medication. The researchers looked at data from 277 patients with HoFH who had been enrolled in three previous clinical trials. These patients had been taking lomitapide for the past several years. The range of data available from these sources enabled the evaluation of follow-up for more than 9 years.


The results of the clinical study showed that long-term treatment with lomitapide was generally safe for the liver. Specifically, the researchers found that the bilirubin levels (a marker of liver function) were not raised in these patients. In addition, the majority of patients had stable or improved liver function tests over the course of the study. There were no clinically relevant increases among hepatic biomarkers CK-18 and CK-18 fragments. Also, there was no meaningful increase in hepatic fat accumulation and liver stiffness among these patients.

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The data shows that lomitapide results in some hepatic alterations, but that overall, there were no clinically meaningful signals of liver damage. These findings are important for individuals with HoFH and their healthcare providers, as they provide reassurance that long-term treatment with lomitapide is unlikely to cause significant liver damage.


The clinical study provides important insights into the long-term hepatic safety of lomitapide in individuals with HoFH. The findings suggest that the medication is generally safe for the liver over extended periods of use, which is reassuring for patients and healthcare providers. However, it's important to remember that all medications come with risks and side effects and that close monitoring is necessary to ensure safe and effective treatment. If you have any concerns about lomitapide or its use in treating HoFH, be sure to speak with your healthcare provider.
Liver International, Dec-15-22
ClinicalTrials.gov NCT00730236

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