Evolocumab for Familial Hypercholesterolemia Promising in Two Studies

(HealthDay News) — Evolocumab shows promise for patients with heterozygous or homozygous familial hypercholesterolemia, according to two studies published online October 1 in The Lancet.

Frederick J. Raal, PhD, from the University of Witwatersrand in Johannesburg, South Africa, and colleagues conducted a multicenter study involving 331 patients with heterozygous familial hypercholesterolemia on stable lipid-lowering therapy for at least four weeks. Participants were randomized to four treatment groups: 140mg evolocumab every two weeks (111 patients), 420mg evolocumab monthly (110 patients), placebo every two weeks (55 patients), or placebo monthly (55 patients). The researchers found that there were significant reductions in the mean low-density lipoprotein (LDL) cholesterol at week 12 with both dosing schedules of evolocumab vs. placebo. Evolocumab was well tolerated, with adverse events at a similar rate to placebo.

In a second study, Raal and colleagues conducted a phase 3 trial at 17 sites in 10 countries involving 49 patients with homozygous familial hypercholesterolemia on stable lipid-regulating therapy. Participants were randomized to evolocumab 420mg (33 patients) or placebo (16 patients) every four weeks. The researchers found that evolocumab significantly reduced ultracentrifugation LDL cholesterol by week 12 by 30.9% compared with placebo (P<0.001). In 63 and 36% of the placebo and evolocumab groups, respectively, treatment-emergent adverse events occurred.

“In patients with homozygous familial hypercholesterolemia receiving stable background lipid-lowering treatment and not on apheresis, evolocumab 420mg administered every four weeks was well tolerated and significantly reduced LDL cholesterol compared with placebo,” the authors write in the second study.

Several authors from both studies disclosed financial ties to pharmaceutical and biotechnology companies, including Amgen, which manufactures evolocumab and funded the study.

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