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Preprint WatchMildSeptember 26th, 2026

Evinacumab Enables Discontinuation of LDL Apheresis in Homozygous Familial Hypercholesterolemia: A Case Report

García Aguilar H, Fokine Dotsenko AGS, Vazquez-Del Campo AR, Gutiérrez–Múgica H, González M.

Evinacumab, an ANGPTL3 inhibitor, produced sustained LDL-C reduction sufficient to discontinue 20 years of LDL apheresis in a single adult HoFH patient, presented as evidence that ANGPTL3 inhibition can replace apheresis in this population.

Mild contradiction

1 prior failure

One documented clinical failure (Phase 1 or 2) overlaps with the claimed mechanism.

This preprint argues from a single case that ANGPTL3 inhibition is sufficient to replace LDL apheresis in homozygous familial hypercholesterolemia. Claidex holds one prior failure against the same target in the same indication, aro-ang3-zodasiran-hofh-phase2-strategic-shutdown, where the ARO-ANG3 (zodasiran) Phase 2 program was stopped by sponsor decision rather than for lack of pharmacology. The two are compatible on mechanism and divergent on what they establish. The approved antibody evinacumab and an RNA interference agent hit the same node by different modalities, so the prior stop says little about ANGPTL3 biology and a great deal about program economics in a rare indication. Severity is MILD because the recorded archetype is a sponsor decision, not an efficacy failure, and because an uncontrolled single-patient report cannot carry the weight the closing sentence places on it.

Abstract excerpt

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) levels and early cardiovascular disease. Despite intensive lipid-lowering therapy, achieving target LDL-C levels remains challenging, and many patients require lifelong LDL apheresis. We report a 33-year-old Mexican–Lebanese woman with confirmed HoFH and multiple comorbidities, including systemic lupus erythematosus. Severe hypercholesterolemia was evident from early childhood, with total cholesterol levels of approximately 800 mg/dL and development of xanthomas at age 3. The patient underwent long-term multimodal therapy, including statins, ezetimibe, PCSK9 inhibitors, and LDL apheresis initiated at age 9 and continued for approximately 20 years, achieving only partial lipid control. Evinacumab, an angiopoietin-like protein 3 (ANGPTL3) inhibitor, was initiated in adulthood as adjunctive therapy. Within two months, significant and sustained LDL-C reduction was achieved, allowing complete discontinuation of LDL apheresis. Follow-up demonstrated stable LDL-C levels between 180–225 mg/dL. This case highlights the clinical effectiveness of Evinacumab in patients with HoFH, demonstrating its potential to achieve sustained lipid reduction and eliminate the need for invasive apheresis. ANGPTL3 inhibition may represent a transformativ

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This is an automated contradiction flag, not an editorial judgment on the preprint's quality. Flags identify where the preclinical literature and the clinical failure record diverge.