Evinacumab May Reduce Apheresis Eligibility in HOFH

In patients with HOFH, evinacumab therapy is associated with decreased risk for apheresis eligibility.

Evinacumab is associated with a significant decrease in qualification for apheresis, compared with baseline, in patients with homozygous familial hypercholesterolemia (HOFH), according to study results presented at the National Lipid Association (NLA) Scientific Sessions 2023, held in Atlanta, Georgia, between June 1 and June 4, 2023.

Researchers presented post-hoc findings regarding the effect of evinacumab on changes in apheresis eligibility. Apheresis eligibility was determined via the 2019 US Food and Drug Administration threshold criteria in patients with HOFH. The single-arm, open-label, phase 3 study (ClinicalTrials.gov Identifier: NCT03409744) enrolled patients aged 12 years or older with HOFH and assessed the long-term tolerability and safety of evinacumab.

The study included a run-in period of up to 10 weeks, a 2-week screening period, an open-label treatment period, and a 24-week follow-up after the last dose of evinacumab. The participants received intravenous evinacumab 15 mg/kg every 4 weeks. The primary endpoint was the severity and incidence of treatment-emergent adverse events (TEAEs) during the open-label treatment period.

A total of 106 patients (mean age, 39 [SD, 16.4] years; 50% men; 71% White) were included. At baseline, 67.9% of patients qualified for lipoprotein apheresis and 32.1% of patients did not per the US eligibility criteria. At week 56, the mean decrease in low-density lipoprotein cholesterol was 130.6 (SD, 109.3) mg/dL from baseline.

Among the 72 patients who qualified for apheresis at baseline, 39 out of 63 patients (data were missing for 9 patients) no longer qualified for apheresis after 56 weeks of treatment. The decrease in the proportion of patients who qualified for apheresis was maintained to week 184 and through the follow-up per protocol. Evinacumab was generally well tolerated, and no new safety signals were observed.

At least 1 TEAE was reported in 81.1% of patients and 19.8% of participants had 1 or more serious TEAE. A TEAE leading to death occurred in 2 patients, and 3 patients had 1 or more TEAE leading to treatment discontinuation. Nasopharyngitis (24.5%) and headache (18.9%) were the most common TEAEs.

Disclosure: This analysis was funded by Regeneron Pharmaceuticals, Inc. Some of the study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


Raal FJ, Rosenson RS, Moriarty PM, et al. Evinacumab reduces US apheresis eligibility in patients with homozygous familial hypercholesterolemia. Abstract presented at: National Lipid Association (NLA) Scientific Sessions 2023; June 1-4, 2023; Atlanta, GA. Abstract #129