Long-term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia: Five-year data from the Lomitapide Observational Worldwide Evaluation Registry (LOWER)

By Underberg JA, Cannon CP, Larrey D, et al
Published September 6, 2020

Key Takeaways

The long-term safety, tolerability, as well as effectiveness of lomitapide (a lipid-lowering agent) was evaluated in an international, observational registry, the Lomitapide Observational Worldwide Evaluation Registry, given that lomitapide is indicated as adjunct treatment for homozygous familial hypercholesterolemia in adults. Researchers assessed 5-year data from the registry up to February 28, 2019. Posttreatment, a mean 33% decrease in low-density lipoprotein cholesterol (LDL-C) was evident, as well as LDL-C <100 mg/dL achievement in 65.4% and LDL-C <70 mg/dL attainment in 41.1%. The absolute mean alteration in LDL-C at year 4 from baseline was found to be –70.6 ± 76.21 mg/dL. The observed efficacy as well as safety of lomitapide was identified to be consistent with phase III trial data, however, a much lower median dose of 10 mg was used compared with 40 mg in phase III. There were no new safety signals. Compared with the phase III trial, lower incidence of adverse events (AEs), serious AEs, and aminotransferase alanine transaminase rises was noted, potentially associated with the lower median dose.

Read the full article on Journal of Clinical Lipidology.

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