Lipid-efficacy RCT · N Engl J Med 2020
ELIPSE HoFH trial
Evinacumab for Homozygous Familial Hypercholesterolemia.
The bottom line
In patients with homozygous familial hypercholesterolemia already on maximal lipid-lowering therapy, adding evinacumab markedly lowered LDL cholesterol compared with placebo, with similar adverse event rates.
Primary lipid endpoint metUS status: US-approved
At a glance
- Medicine
- Evinacumab 15 mg per kilogram intravenous infusion every 4 weeks
- Compared with
- Placebo (intravenous infusion every 4 weeks)
- People
- 65
- Who took part
- Patients with homozygous familial hypercholesterolemia receiving stable lipid-lowering therapy; mean baseline LDL cholesterol 255.1 mg per deciliter despite maximum doses of background lipid-lowering therapy
- Design
- Double-blind, placebo-controlled, phase 3 trial, randomized 2:1
- Follow-up
- 24 weeks
- Main question
- Percent change from baseline in LDL cholesterol level at week 24
Main result
Between-group least-squares mean difference in percent change in LDL cholesterol of -49.0 percentage points (95% CI, -65.0 to -33.1; P<0.001)
Effect on LDL cholesterol
Relative reduction from baseline in LDL cholesterol of 47.1% with evinacumab vs. increase of 1.9% with placebo; between-group least-squares mean difference -49.0 percentage points (95% CI, -65.0 to -33.1; P<0.001); absolute difference -132.1 mg per deciliter (95% CI, -175.3 to -88.9; P<0.001)
Other results
LDL cholesterol was lower with evinacumab than placebo in patients with null-null variants (-43.4% vs. +16.2%) and in those with non-null variants (-49.1% vs. -3.8%)
Safety
Adverse events were similar in the two groups.
In the trial's own words
“At week 24, patients in the evinacumab group had a relative reduction from baseline in the LDL cholesterol level of 47.1%, as compared with an increase of 1.9% in the placebo group, for a between-group least-squares mean difference of -49.0 percentage points (95% confidence interval [CI], -65.0 to -33.1; P<0.001); the between-group least-squares mean absolute difference in the LDL cholesterol level was -132.1 mg per deciliter (95% CI, -175.3 to -88.9; P<0.001).”
Quoted from the abstract of N Engl J Med 2020, checked word for word against PubMed. PubMed 32813947 · Full paper (DOI)
How this medicine works, where it stands and what it costs: Evinacumab (Evkeeza)
Common questions
What did the ELIPSE HoFH trial find?
In patients with homozygous familial hypercholesterolemia already on maximal lipid-lowering therapy, adding evinacumab markedly lowered LDL cholesterol compared with placebo, with similar adverse event rates.
Who was in ELIPSE HoFH?
65 people. Patients with homozygous familial hypercholesterolemia receiving stable lipid-lowering therapy; mean baseline LDL cholesterol 255.1 mg per deciliter despite maximum doses of background lipid-lowering therapy.
What was ELIPSE HoFH's main result?
Between-group least-squares mean difference in percent change in LDL cholesterol of -49.0 percentage points (95% CI, -65.0 to -33.1; P<0.001)
How much did LDL cholesterol change in ELIPSE HoFH?
Relative reduction from baseline in LDL cholesterol of 47.1% with evinacumab vs. increase of 1.9% with placebo; between-group least-squares mean difference -49.0 percentage points (95% CI, -65.0 to -33.1; P<0.001); absolute difference -132.1 mg per deciliter (95% CI, -175.3 to -88.9; P<0.001)
Related trials
- Evinacumab refractory HC (2020)In patients with refractory hypercholesterolemia despite maximal lipid-lowering therapy, evinacumab substantially and significantly lowered LDL cholesterol compared with placebo, with serious adverse events occurring at variable rates across dose groups.
- APPROACH (2019)In patients with familial chylomicronemia syndrome, volanesorsen substantially lowered triglyceride and apolipoprotein C-III levels compared with placebo but caused frequent injection-site reactions and thrombocytopenia.
- ARCHES-2 (2024)In adults with mixed hyperlipidemia, zodasiran (an RNAi therapy against ANGPTL3) produced significant, dose-dependent reductions in triglycerides and improvements in non-HDL-C, ApoB, and LDL-C compared with placebo, with a signal of transient HbA1c elevation in diabetic patients at the highest dose.
- Balance (2024)In patients with familial chylomicronemia syndrome, olezarsen 80 mg significantly lowered triglycerides and markedly reduced acute pancreatitis episodes compared with placebo, with the 50-mg dose not reaching significance for the triglyceride endpoint.
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