Lomitapide Effective in Lowering LDL Cholesterol in Children with Homozygous Familial Hypercholesterolemia: APH-19 Study
Spain: A recent phase 3 study, published in The Lancet Diabetes & Endocrinology, has demonstrated the effectiveness of Lomitapide in treating pediatric patients with homozygous familial hypercholesterolemia (HoFH). This rare genetic condition leads to dangerously high levels of low-density lipoprotein (LDL) cholesterol.
The multicenter, open-label trial, APH-19, revealed significant reductions in LDL cholesterol levels, offering a promising new option for managing this challenging condition in children.
HoFH is characterized by genetic mutations that hinder the ability of the body to process cholesterol effectively, often resulting in severe cardiovascular complications at a young age. Traditional treatments, such as statins, may not provide adequate relief due to the unique metabolic challenges faced by these patients, making alternative therapies crucial.
Lomitapide is an orally administered inhibitor of microsomal triglyceride transfer protein (MTP) that effectively reduces LDL cholesterol levels and is approved for use in adults with homozygous familial hypercholesterolemia. Considering this, Prof Luis Masana, Sant Joan University Hospital, CIBERDEM, Reus, Spain, and colleagues sought to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who were receiving standard lipid-lowering therapy.
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