National Lipid Association

National Lipid Association

June 01, 2017
2 min read

Evinacumab plus lipid-lowering therapy may benefit patients with homozygous familial hypercholesterolemia

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PHILADELPHIA — Patients with homozygous familial hypercholesterolemia treated with evinacumab in addition to lipid-lowering therapy had significant decreases in LDL, according to a poster presented at National Lipid Association Scientific Sessions.

Researchers analyzed data from nine patients with homozygous familial hypercholesterolemia (mean LDL, 376 ± 240.9 mg/dL) who were receiving lipid–lowering therapy; none had lipoprotein apheresis within 4 weeks of screening. Open–label treatment with the novel agent evinacumab (Regeneron) and follow–up were conducted for 26 weeks.

“These results are suggestive of an add-on contribution of evinacumab (over that of current [lipid-lowering therapies]) for inhibition of ANGPTL3 in the treatment of [homozygous familial hypercholesterolemia], with the potential for LDL normalization in some patients presenting extreme baseline LDL levels,” Daniel Gaudet, MD, PhD, professor of medicine at the University of Montreal, and colleagues wrote.

Daniel Gaudet

The primary endpoint was percent change in LDL from baseline to 4 weeks. Key secondary endpoints included the following:

  • Percent and absolute change in LDL from week 2 to week 4,
  • Percent and absolute change in LDL from baseline over time,
  • Absolute change in LDL from baseline to week 4,
  • Percent and absolute change in apolipoprotein B, total cholesterol, non–HDL and lipoprotein(a) from baseline over time, and
  • Severity and prevalence of adverse events.

At 4 weeks, evinacumab lowered LDL by 49 ± 23% (range, 25-90), with peak reduction of –58 ± 18% transpiring between weeks 4 and 12. All patients experienced significant LDL reductions. Evinacumab also lowered HDL (–36 ± 16%), triglycerides (–47%; interquartile range [IQR], –57 to –38), total cholesterol (–47 ± 19%), ApoB (–46 ± 18%) and lipoprotein(a) (–19%; IQR, –27 to 1).

Although no patients were hypertriglyceridemic at baseline (IQR, 55-105 mg/dL), median plasma triglyceride levels were reduced by 47%.

One patient developed CAD from an underlying disease, but it was not related to evinacumab. Treatment-emergent adverse events including myalgia (n = 1), mild injection site reactions (n = 2), epistaxis (n = 1) and mild hot flush (n = 2) occurred, but did not lead to treatment discontinuation.

“Our results suggest that evinacumab may have potential as a treatment of [homozygous familial hypercholesterolemia] in patients currently on [lipid-lowering therapy] or lipoprotein apheresis,” Gaudet and colleagues wrote. – by Darlene Dobkowski


Gaudet D, et al. Abstract 204. Presented at: National Lipid Association Scientific Sessions; May 18–21, 2017; Philadelphia.

Disclosure: The study was funded by Regeneron Pharmaceuticals. Gaudet reports serving as a consultant/advisory panel member for Aegerion, Amgen, Catabasis, Chiesi, Cymabay, Gemphire, Ionis, Novartis, Omthera, Regeneron, Sanofi and Uniqure.