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Lomitapide

(Juxtapid®)

Juxtapid®

Drug updated on 12/11/2024

Dosage FormCapsule (oral; 5 mg, 10 mg, 20 mg, 30 mg)
Drug ClassMicrosomal 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
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  • This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
  • Lomitapide demonstrated significant low-density lipoprotein cholesterol (LDL-C) reduction in homozygous familial hypercholesterolemia (HoFH) patients across both pediatric and adult populations, as shown in 18 clinical studies involving 120 patients; clinical effects in children mirrored those observed in adults.
  • PCSK9 inhibitors were reported to be safe and moderately effective for LDL-C reduction in HoFH patients across two randomized controlled trials (RCTs), while Evinacumab showed safety and effectiveness across all HoFH subgroups in an RCT.
  • Ezetimibe combined with a statin demonstrated moderate LDL-C reduction in an RCT, and LDL apheresis, although effective, had evidence marked by a high risk of bias.
  • Lomitapide was associated with gastrointestinal disturbances and elevated hepatic alanine aminotransferase (ALT) levels in HoFH patients, with most adverse events managed through dietary and dose adjustments, allowing for general tolerability across both pediatric and adult populations.
  • PCSK9 inhibitors and Evinacumab demonstrated a favorable safety profile in randomized controlled trials (RCTs), while specific safety data for Ezetimibe with Statin and LDL Apheresis were not detailed for direct comparison.
  • There is no population types or subgroups information available in the reviewed studies.