01 Apr Monoclonal Antibody Evinacumab is a Major Benefit to Patients with Homozygous Familial Hypercholesterolemia

MedicalResearch.com Interview with:

Professor F. J. Raal

Professor F. J. Raal, FRCP, FCP(SA), Cert Endo, MMED, PhD
Director, Carbohydrate & Lipid Metabolism Research Unit
Professor & Head, Division of Endocrinology & Metabolism,
Faculty of Health Sciences, University of the Witwatersrand

MedicalResearch.com: What is the background for this study? How does Evinacumab differ from the three drugs used in triple therapy for this severe form of hypercholesterolemia?

Response:      Despite available lipid lowering therapies, the vast majority of patients with homozygous familial hypercholesterolemia are unable to achieve desirable LDL-cholesterol levels and remain at high risk for premature atherosclerotic cardiovascular disease.

Unlike statins and PCSK9-inhibitors which act mainly by upregulating LDL receptor activity on the cell surface, evinacumab, a monoclonal antibody inhibitor of ANGPTL3, acts independent of the LDL receptor.

MedicalResearch.com: What are the main findings?

Response:     In this phase III study in 65 subjects with homozygous familial hypercholesterolemia  randomized to evinacumab or placebo, evinacumab reduced LDL-cholesterol by 49% compared to placebo, an absolute reduction in LDL-cholesterol of 132 mg/dL (3.4 mmol/L)  which is remarkable. The drug was equally effective in those homozygous familial hypercholesterolemia patients with minimal or no residual LDL receptor function.

MedicalResearch.com: What should readers take away from your report?

Response: Evinacumab can substantially reduce LDL-cholesterol levels in homozygous familial hypercholesterolemia patients regardless of LDL receptor function and is an effective treatment option for these high risk patients who are unable to reach LDL-cholesterol target despite multiple lipid-lowering therapies with or without apheresis. 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: The addition of evinacumab is of major benefit to patients with homozygous familial hypercholesterolemia as it halves their LDL-cholesterol levels and with ongoing treatment, provided there are no long-term safety issues, this is likely to markedly reduce their risk for premature cardiovascular disease and improve survival.

MedicalResearch.com: Is there anything else you would like to add?

Response: With the addition of evinacumab to the other lipid lowering therapies available (statins, ezetimibe plus PCSK9 inhibitors) we will change the natural history of homozygous familial hypercholesterolemia from one that is often lethal in childhood to a manageable and treatable lipid disorder.

My disclosures: I have received research grants, honoraria, or consulting fees for professional input and/or delivered lectures from Sanofi, Regeneron, Amgen and The Medicines Company. 

Citation:

Raal F. Evinacumab in patients with homozygous familial hypercholesterolemia. Presented on: March 30, 2020. ACC 2020.

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Last Updated on April 1, 2020 by Marie Benz MD FAAD