Homozygous familial hypercholesterolemia (HoFH) is a rare and severe condition associated with extremely high cholesterol levels and significant cardiovascular morbidity and mortality at an early age during adolescence. Despite combination therapy with multiple lipid-lowering drugs, including PCSK9 inhibitors, the majority of affected patients also require lifelong, time-and resource-intensive lipoprotein apheresis. Evinacumab is a new, innovative lipid-lowering agent whose mechanism of action is independent of the LDL receptor. It has been approved in 2021 as an adjuvant therapy for reducing LDL-C in adults and children with HoFH, filling a crucial gap in the treatment landscape for the disease. Data from several randomized controlled trials, as well as real-world observational studies, have confirmed the safety and effectiveness of monthly intravenous evinacumab administration over treatment periods spanning several years. On average, when used alongside established guideline-based lipid-lowering combination therapy (including PCSK9 inhibitors), it results in an LDL-C reduction of about 45-50%. In some cases, this has allowed patients to discontinue lipoprotein apheresis. Evinacumab thus opens up a promising new perspective in the management of HoFH. J Kardiol 2026; 33: (1-2): 11-6.
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