Key result
Lomitapide reduces LDL-C by ~50% in patients with homozygous familial hypercholesterolemia.
Why the study?
Does lomitapide reduce LDL-C in patients with homozygous familial hypercholesterolemia?
Does lomitapide reduce LDL-C in patients with homozygous familial hypercholesterolemia?
Answers are grounded in this paper and cite their sources.
Lomitapide effectively reduces LDL-C by 50% at 26 weeks in patients with homozygous familial hypercholesterolemia.
omozygous familial hypercholesterolemia is a genetic disorder characterized by low-density lipoprotein (LDL)-receptor dysfunction, markedly elevated levels of LDL-cholesterol (LDL-C) and premature atherosclerosis. Patients are often poorly responsive to conventional lipid-lowering therapies that upregulate LDL-receptor expression. In a pivotal 78-week openlabel trial, lomitapide, titrated to the maximal tolerable dose, decreased LDL-C by 50% at the end of the efficacy phase (week 26) in patients with homozygous familial hypercholesterolemia.
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Blom et al. (2017) studied Homozygous familial hypercholesterolemia. Lomitapide was evaluated on LDL-C reduction (50% decrease). Lomitapide titrated to the maximal tolerable dose decreased LDL-C by 50% at the end of the 26-week efficacy phase in patients with homozygous familial hypercholesterolemia.
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