Santos RD, Stein EA, Hovingh GK, et al. - Researchers tested long-term safety as well as efficacy of evolocumab as a treatment option for patients with homozygous familial hypercholesterolemia (FH) (HoFH) and severe heterozygous FH (HeFH) in this open-label, single-arm study. Subcutaneous evolocumab 420 mg monthly or 420 mg every 2 weeks was initiated in patients with HoFH or severe HeFH ≥ 12 years of age and on stable lipid-lowering therapy if they were receiving lipoprotein apheresis. Following 12 weeks, up-titration to 420 mg every 2 weeks could be done in those not on apheresis. Nasopharyngitis, influenza, upper respiratory tract infection, and headache were the adverse events that were experienced most commonly. The occurrence of adverse events in 89.3% of patients was documented. Overall, findings revealed good tolerability as well as the effectiveness of evolocumab in decreasing plasma low-density lipoprotein cholesterol levels in patients with HoFH and severe HeFH over a median of 4.1 years.