Drug updated on 9/4/2024
| 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).
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Summary
- Juxtapid (lomitapide) 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 (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
- This summary is based on the review of three systematic review(s)/meta-analysis(es). [1-3]
- LDL-C Reduction: In 39 Japanese patients with homozygous familial hypercholesterolemia (HoFH), mean LDL-C levels significantly decreased from 225.9 ± 172.0 mg/dl (5.8 ± 4.5 mmol/l) to 159.4 ± 93.0 mg/dl (4.1 ± 2.4 mmol/l) at 12 months (p = 0.0245).
- LDL-C Reduction: Lomitapide significantly reduced LDL-C levels in 120 HoFH patients, with comparable effects observed between pediatric and adult populations.
- Comparative Efficacy: Evolocumab and Alirocumab were the most effective nonstatin lipid-lowering therapies compared to Lomitapide in HoFH patients, with other regimens including Inclisiran and Bempedoic acid/ezetimibe also showing efficacy. 6. 61.5% of patients (24 out of 39) experienced 74 drug-related adverse events, with liver-related events occurring in 35.9%, gastrointestinal disorders in 48.7%, and one bleeding disorder (2.6%). Lomitapide dose adjustments were required for 39.2% of these events, temporary withdrawal in 12.2%, and permanent discontinuation in 1.4%.
- The most common adverse events reported were gastrointestinal disturbances and elevated hepatic ALT levels. These events were generally manageable through low-fat diets and dose adjustments, with no significant differences in safety profiles between pediatric and adult HoFH patients.
- There is no population types or subgroups information available in the reviewed studies.
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 |