Real-world evaluation of bempedoic acid use in patients with homozygous familial hypercholesterolemia.

OBJECTIVE

Evaluate efficacy and tolerability of bempedoic acid in homozygous familial hyper cholesterolemia (HoFH).

METHODS

A single-center retrospective study of HoFH patients prescribed bempedoic acid between February 2020 and May 2025. Patients were managed according to our clinical standard of care, with routine assessments of lipids, medication tolerability, and adherence. The primary endpoint was percent change in low-density lipoprotein cholesterol (LDL-C) from baseline to first follow-up. Secondary endpoints evaluated LDL-C changes at other timepoints, LDL-C goal attainment, medication access, and tolerability.

RESULTS

Of the 14 HoFH patients treated within our clinic, 5 (mean age 41.4 years, 60% female) were initiated on bempedoic acid, and 4 continued treatment. Pre-existing atherosclerotic cardiovascular disease (ASCVD) was present in 4 of 5 patients, and baseline mean ± SD LDL-C was 131 ± 62.4 mg/dL. Patients were managed on a mean of 3.8 lipid-lowering therapies at baseline, all with statins (60% high-intensity). Bempedoic acid reduced LDL-C by a mean ± SD of -33.2% ± 15.1% to an on-treatment LDL-C of 71 ± 30.9 mg/dL at first follow-up (median 2.9 months). LDL-C reduction of -29.4% ± 13.1% to an on-treatment LDL-C of 75 ± 29.6 mg/dL was seen at most recent follow-up (median 25.6 months). Two patients achieved recommended LDL-C goals on bempedoic acid. The medication was well tolerated, with 60% free from adverse events.

CONCLUSION

Bempedoic acid in patients with HoFH was both effective for LDL-C lowering and tolerable in this cohort of patients. This represents a novel treatment approach in a very high-risk patient population requiring multi-drug regimen for lipid optimization and cardiovascular protection.