Efficacy and Safety of Evinacumab in Japanese Patients with Homozygous Familial Hypercholesterolemia: Long-term Results from an Open-label, Single-arm, Phase 3 Trial.

Authors

Masahiro Koseki, Hisashi Makino, Yoshihiko Otsubo, Masa-Aki Kawashiri, Naohiko Fujii, Naoya Fujita, Yukiyo Yamamoto, Alpana Waldron, Robert Pordy, Shazia Ali, Serap Sankoh, Pinay Kainth, Hayato Tada, Kimimasa Tobita, Mariko Harada-Shiba

Published in

Journal of atherosclerosis and thrombosis. Jul 01, 2026. Epub Jul 01, 2026.

Abstract

This sub-group analysis evaluated the long-term efficacy and safety of evinacumab, an angiopoietin-like 3 inhibitor, in adolescent and adult Japanese patients with homozygous familial hypercholesterolemia (HoFH) who were enrolled in the open-label, single-arm, extension of the Phase 3 ELIPSE HoFH pivotal trial.
Patients aged ≥ 12 years with HoFH on stable lipid-lowering therapies received intravenous evinacumab 15 mg/kg every 4 weeks and weekly/biweekly lipoprotein apheresis. Final results are reported.
Eleven patients were enrolled and received at least 1 dose of evinacumab at baseline, including 9 adults who participated in the evinacumab pivotal study (evinacumab-continue) and 2 newly enrolled adolescent patients (evinacumab-naïve). Mean (standard error) percent decrease in LDL-C from baseline to Week 24 was 42.9% (7.4) in the overall cohort (n = 9) and 37.1% (7.9) in the evinacumab-continue group (n = 7), with reductions in LDL-C observed as early as Week 8 and persisting through Week 168. Evinacumab treatment was also associated with reductions in other lipid parameters measured, including apolipoproteins A1 and B, non-high density lipoprotein cholesterol (HDL-C), HDL-C, total cholesterol, fasting triglycerides, and lipoprotein(a). Similar lipid-lowering results were seen in the evinacumab-naïve group (n = 2). Treatment-emergent adverse events (TEAEs) occurred in all 11 patients. Serious TEAEs occurred in 3 (27.3%) patients, and none were treatment related.
Evinacumab treatment resulted in sustained reductions in LDL-C and other lipid parameters in Japanese patients ≥ 12 years with HoFH, including those undergoing lipoprotein apheresis, and was generally well tolerated.

PMID:
42386604
Bibliographic data and abstract were imported from PubMed on 02 Jul 2026.

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