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Two studies demonstrate LDL-lowering with evolocumab in patients with familial hypercholesterolemia

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Confirming findings presented at recent scientific meetings, the PCSK9 inhibitor evolocumab was associated with reduced LDL levels and a low rate of adverse events in patients with familial hypercholesterolemia, according to data from two studies published in The Lancet.

Frederick J. Raal, MD, from the carbohydrate and lipid metabolism research unit of the faculty of health sciences at the University of Witwatersrand in Johannesburg, and colleagues conducted the RUTHERFORD-2 study of evolocumab (Amgen) in patients with heterozygous familial hypercholesterolemia (n=331) and the TESLA Part B study in patients with homozygous familial hypercholesterolemia (n=49).

In the RUTHERFORD-2 study, patients were randomly assigned evolocumab 140 mg every 2 weeks, evolocumab 420 every month, placebo every 2 weeks or placebo every month.

Compared with placebo, the evolocumab groups had reduced mean LDL at 12 weeks (every 2 weeks dose, 59.2% reduction; 95% CI, 53.4-65.1; every month dose, 61.3% reduction; 95% CI, 53.6-69). The researchers observed a similar LDL reduction at the mean of 10 and 12 weeks.

Adverse event rates were similar between evolocumab and placebo. Nasopharyngitis (9% vs. 5%) and muscle-related adverse events (5% vs. 1%) occurred more frequently in the evolocumab group.

In the TESLA Part B study, patients were randomly assigned evolocumab 420 mg monthly or placebo for 12 weeks.

At 12 weeks, compared with the placebo group, the evolocumab group had reduced ultracentrifugation LDL by 30.9% (95% CI, –43.9 to –18), while no serious clinical or laboratory adverse events occurred, Raal and colleagues wrote.

In a related editorial, Raul D. Santos, MD, PhD, MSc, and Gerald F. Watts, DSc, PhD, DM, FRACP, DRCP, noted that despite the short duration of RUTHERFORD-2 and TESLA Part B, the results “are compatible with longer-term observations in other groups of patients with hypercholesterolemia and they provide good-quality data that will advance the future care of familial hypercholesterolemia.”

Further, “if proven to be safe and efficacious in the long term, as well as cost effective, PCSK9 monoclonal antibodies might be the best standard of care for many patients with severe forms of familial hypercholesterolemia,” Santos, from the Lipid Clinic, Heart Institute (InCor), University of Sao Paolo Medical School Hospital, and Watts, from the Lipid Disorders Clinic, Royal Perth Hospital School of Medicine and Pharmacology, University of Western Australia, wrote. “This treatment could also apply to a wider range of higher-risk patients with polygenic hypercholesterolemia, including those with statin myopathy.”

For more information:

Raal FJ. Lancet. 2014;doi:10.1016/S0140-6736(14)61374-X.

Raal FJ. Lancet. 2014;doi:10.1016/S0140-6736(14)61399-4.

Santos RD. Lancet. 2014;doi:10.1016/S0140-6736(14)61702-5.

Disclosure: The studies were funded by Amgen. See the full studies for a list of the researchers’ relevant financial disclosures. See the full editorial for a list of Santos’ and Watts’ relevant financial disclosures.