Drug updated on 4/15/2024
| Dosage Form | Capsule (oral; 5 mg, 10 mg, 20 mg, 30 mg) |
| Drug Class | Microsomal triglyceride transfer protein inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
Summary
- Lomitapide (Juxtapid) is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia.
- Two studies were analyzed. These studies provided information regarding the safety and effectiveness of Lomitapide compared to other nonstatin lipid-lowering therapies.
- In a study from Japan focusing on patients with homozygous familial hypercholesterolemia over 42 months, Lomitapide effectively reduced mean LDL-C levels from 225.9 mg/dl predose to 159.4 mg/dl at 12 months, despite adverse events such as gastrointestinal disorders and liver-related issues being reported by a significant portion of patients.
- Compared with other nonstatin therapies added to maximally tolerated statins in populations at high cardiovascular risk, not limited only to genetic conditions like HoFH but also elevated LDL-C levels, evolocumab and alirocumab were the most efficacious regimens in reducing LDL-C at both week 12 and week 24. Bempedoic acid/ezetimibe and ezetimibe monotherapy demonstrated effectiveness but weren't as potent when compared against PCSK9 inhibitors.
- The evaluation of Lomitapide's safety focused on individuals with HoFH, a severe genetic disorder leading to significantly elevated cholesterol levels, providing valuable insight into its performance outside controlled trials within this high-risk population group.
- Lomitapide appears effective for lowering LDL-C in patients suffering from homozygous familial hypercholesterolemia, considering the severity of the condition. However, it requires careful monitoring due to its safety profile marked by gastrointestinal and liver-related adverse events.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Juxtapid (lomitapide) Prescribing Information. | 2020 | Amryt Pharmaceuticals DAC, Dublin, Ireland |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan. | 2024 | Future Cardiology |
| Network meta-analysis of randomized trials evaluating the comparative efficacy of lipid-lowering therapies added to maximally tolerated statins for the reduction of low-density lipoprotein cholesterol. | 2022 | Journal of The American Heart Association |
| Efficacy and safety of lomitapide in homozygous familial hypercholesterolaemia: a systematic review. | 2022 | Reviews in Cardiovascular Medicine |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Contemporary lipid-lowering management and risk of cardiovascular events in homozygous familial hypercholesterolaemia: insights from the Italian LIPIGEN Registry. | 2024 | European Journal of Preventive Cardiology |
| Updates in the management of pediatric dyslipidemia. | 2023 | Current Opinion in Lipidology |
| Advancements in the treatment of homozygous familial hypercholesterolemia. | 2022 | Journal of Atherosclerosis and Thrombosis |
| Beyond statins and PCSK9 inhibitors: updates in management of familial and refractory hypercholesterolemias. | 2021 | Current Cardiology Reports |
| Lipid-Lowering Drugs. | 2019 | The Medical Letter |