Key result
Bempedoic acid lowers LDL-C up to ~21% versus placebo regardless of HeFH status.
Why the study?
Patients with heterozygous familial hypercholesterolemia often cannot reach guideline-recommended LDL-C goals despite multidrug therapy.
Does bempedoic acid reduce LDL-C in patients with heterozygous familial hypercholesterolemia receiving maximally tolerated statin therapy?
RCT (n=3,009)
2:1
Yes
Does bempedoic acid reduce LDL-C in patients with heterozygous familial hypercholesterolemia receiving maximally tolerated statin therapy?
Effect estimate: -21.2% (with HeFH) and -18.2% (without HeFH) vs placebo
p-value: p=<0.0001
Bempedoic acid significantly lowers LDL-C levels when added to maximally tolerated statin therapy in patients with heterozygous familial hypercholesterolemia, with a safety profile comparable to placebo.
Supports adding bempedoic acid to max-tolerated statins in HeFH; extends efficacy data to this high-risk population.
BACKGROUND: Patients with heterozygous familial hypercholesterolemia (HeFH) often cannot reach guideline-recommended low-density lipoprotein cholesterol (LDL-C) goals despite multidrug therapy. OBJECTIVE: To evaluate the efficacy and safety of bempedoic acid as an add-on therapy for lowering LDL-C in patients with HeFH. METHODS: Pooled data from two 52-week phase 3 clinical trials of patients with atherosclerotic cardiovascular disease and/or HeFH receiving maximally tolerated statin therapy (randomized 2:1 to bempedoic acid or placebo) were analyzed by HeFH status. Endpoints included changes from baseline to week 12 (and up to week 52) in LDL-C and other lipid parameters, achievement of LDL-C goals, and safety. RESULTS: A total of 217 (bempedoic acid, 146; placebo, 71) patients with HeFH and 2,792 (bempedoic acid, 1,864; placebo, 928) without HeFH were included (mean baseline LDL-C, 172.8 mg/dL and 102.6 mg/dL, respectively). Bempedoic acid significantly lowered LDL-C at week 12 vs. placebo regardless of HeFH status (with HeFH, -21.2%; without HeFH, -18.2% [both P<0.0001]). Bempedoic acid significantly reduced other lipid parameters and high-sensitivity C-reactive protein vs. placebo regardless of HeFH status (all P≤0.01). Among patients with HeFH treated with bempedoic acid, 32% and 27% achieved LDL-C <100 mg/dL at weeks 12 and 52, respectively. Overall treatment-emergent adverse event incidence was comparable across all four groups (74.7-77.5%). CONCLUSION: Bempedoic acid significantly lowered LDL-C levels vs. placebo and was generally well tolerated in all patients, with no new safety findings in patients with HeFH, despite more intensive lipid-lowering therapy in patients with vs. without HeFH.
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Duell et al. (2024) conducted an RCT in Heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (n=3,009). Bempedoic acid vs. Placebo was evaluated on Change from baseline to week 12 in LDL-C (-21.2% (with HeFH) and -18.2% (without HeFH) vs placebo, p=<0.0001). Bempedoic acid significantly lowered LDL-C at week 12 vs. placebo in patients with and without heterozygous familial hypercholesterolemia (-21.2% and -18.2% respectively; P<0.0001).
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