Evinacumab achieves significant LDL-C reduction in difficult-to-treat HoFH

Evinacumab has potential in the treatment of high-risk patients with homozygous familial hypercholesterolemia (HoFH), according to a case report from the first use of evinacumab in an Indian child with HoFH.

Diagnosed at age two, the patient exhibited severe lipid abnormalities due to a homozygous LDL receptor (LDLR) gene deletion. Despite lifestyle modifications, high-dose statins, ezetimibe, and evolocumab, her LDL-C remained high at an average of 320.3 mg/dL. Her caregivers declined LDL-apheresis, and she eventually required aortic valve replacement due to progressive cardiovascular complications.

With the addition of evinacumab to her treatment, her LDL-C levels dropped by 76.16% to 82 mg/dL, a result sustained for 4 months.

Reference
Arora S, Banerjee S, George A, et al. Dramatic response to Evinacumab in a North Indian girl with homozygous familial hypercholesterolemia. J Pediatr Endocrinol Metab. 2025;38(3):305-309. doi: 10.1515/jpem-2024-0506. PMID: 40022592.