Long-term safety and effectiveness of evinacumab in people with homozygous familial hypercholesterolemia: a plain language summary
- PMID: 40546112
- PMCID: PMC12258244
- DOI: 10.1080/14796678.2025.2513805
Long-term safety and effectiveness of evinacumab in people with homozygous familial hypercholesterolemia: a plain language summary
Plain language summary
What is this summary about?This summary explains the results of a study looking at the long-term effects of a treatment called evinacumab. This study included people aged over 12 years with homozygous familial hypercholesterolemia, also known as HoFH. HoFH is a rare genetic disorder that makes the liver less able to remove excess ‘bad’ cholesterol (called low-density lipoprotein cholesterol, or LDL-C) from the body. LDL-C is considered ‘bad’ cholesterol because it increases the risk of heart disease and stroke. High levels of LDL-C increase the risk of developing serious heart problems early in life. Treatment options for lowering LDL-C often do not work very well for people with HoFH. Evinacumab is a medicine that is approved for the treatment of HoFH as an add-on to other therapies that lower LDL-C. The effect of evinacumab on heart problems such as heart attacks, stroke, or death is not known. In the UK, evinacumab is approved for people with HoFH aged 6 months and above. In Canada and the USA, evinacumab is approved for people with HoFH aged 5 years and above. In the European Union/European Economic Area, evinacumab is approved for people with HoFH aged 6 months and above. In Japan, evinacumab is approved for all ages of people with HoFH. More recently, evinacumab has also been approved in Brazil and Israel.What were the results?People with HoFH who took part in the study were treated with evinacumab every 4 weeks for up to 44 months.After 6 months of treatment, LDL-C levels were almost 44% lower than when people started the study.Treatment-emergent adverse events, also called TEAEs, were reported by 80% of people in the study but most of these were mild (23.3%) or moderate (41.4%) in severity. The most commonly reported side effects which happened during the study were common cold, COVID-19, headache, flu-like illness, general pain, back pain, nausea, and cough. The study researchers determined that most of these reported side effects (91%) were not related to the use of evinacumab.No serious TEAEs were considered related to evinacumab treatment. Nearly all of the people (97%) could continue with the study treatment and, based on this, the researchers concluded that evinacumab was generally well tolerated.What do the results mean?This study showed that evinacumab lowered ‘bad’ cholesterol in people with HoFH, and that this result lasted for a long period of time.[Box: see text].
Conflict of interest statement
D.G. reports grants and personal fees from Regeneron Pharmaceuticals, Inc. during the conduct of the study; and research grants, honoraria, or consulting fees for professional input and/or delivered lectures from Amgen, Amryt Pharma, Arrowhead, Eli Lilly, Esperion, Pfizer, Sanofi, Novartis, Ultragenyx, and Verve Therapeutics outside the submitted work. S.G-P. reports receiving research support from Akcea Therapeutics, Amgen, Regeneron Pharmaceuticals, Inc., and Sanofi; lecture fees from Akcea Therapeutics, Chiesi, and Ultragenyx; support for attending meetings and/or travel from Akcea Therapeutics, Chiesi, and Ultragenyx; and receipt of equipment from Abbott Laboratories. L.F.R. reports investigational fees from Regeneron Pharmaceuticals, Inc.; and speaker fees from Ultragenyx, Daiichi Sankyo, and Novartis. G.I. reports research grants from Amgen, Sanofi, and Regeneron Pharmaceuticals, Inc.; consulting fees from Akcea Therapeutics, Amryt Pharma, Daiichi Sankyo, Novartis, Sanofi, and Ultragenyx; fees for educational events from Amryt Pharma and Ultragenyx; advisory board fees from Daiichi Sankyo and Ultragenyx; and support for attending meetings and/or travel from Lusofarmaco and Sanofi. R.S.R. reports research grants and/or personal fees outside the submitted work from Regeneron Pharmaceuticals, Inc., Amgen, Arrowhead, Avilar Therapeutics, CRISPR Therapeutics, Kowa, Lilly, Lipigon, Meda Pharma, Merck, Novartis, Precision BioSciences, UpToDate, UltraGenyx, and Verve Therapeutics; and stock holdings in MediMergent, LLC. S.S. reports consulting fees from Regeneron Pharmaceuticals, Inc., Octapharma France, Argenx France, and Vifor Pharma France. C.S. reports research grants from Akcea Therapeutics, Amgen, Amryt Pharma, B. Braun Avitum, Fresenius Medical Care, Kaneka Pharma Europe, Regeneron Pharmaceuticals, Inc., and Sanofi. E.S. reports grants and/or personal fees outside the submitted work from Amgen, AstraZeneca, Daiichi Sankyo, Ionis Pharmaceuticals, Novo Nordisk, Novartis, and Sanofi. A.W. reports payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing, or educational events from Novartis and Algorithm; participation in a data safety monitoring board or advisory board for Chiesi; leadership or fiduciary role in other board, society, committee, or advocacy group, paid or unpaid, for Novartis; and research support for pharmaceutical trials from Amgen, Regeneron Pharmaceuticals, Inc., Novartis, Silence Therapeutics, Esperion, Sanofi, and Ultragenyx. T.T. reports no conflicts of interest. S.A., P.B., J.M., R.T.G., X-Q.Z., R.P., and J.Z. are employees of and shareholders in Regeneron Pharmaceuticals, Inc. TA.W. is an employee of and shareholder in Regeneron Pharmaceuticals, Inc.; and reports being a shareholder in Bristol Myers Squibb. E.B. reports consulting fees from Aegerion, Akcea Therapeutics, Amarin, Amgen, Genfit, Ionis Pharmaceuticals, Lilly, Merck Sharp & Dohme, Mylan, Novartis, Regeneron Pharmaceuticals, Inc., Sanofi, and Servier. F.J.R. has received research grants, honoraria, or consulting fees for professional input and/or delivered lectures from Amgen, Sanofi-Aventis, Regeneron Pharmaceuticals, Inc., Novartis, LIB Therapeutics, and Ultragenyx. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Writing support for this summary was provided by Alpha (a division of Prime, Knutsford, UK), and was funded by Regeneron Pharmaceuticals, Inc.
Patient reviewers on this PLSP have received honorarium from
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.
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