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For the first time, paediatric patients aged 5-11 years with HoFH in the EU have access to a first-in-class medicine, Evkeeza (evinacumab), to treat an ultrarare, inherited form of high cholesterol

Read time: 1 mins
Published:19th Dec 2023

Ultragenyx Pharmaceutical Inc. announced that the European Commission (EC) has extended the approval of Evkeeza (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH)

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH. Evkeeza initially received EC decision as an adjunct to diet and other lipid-lowering therapies in adolescents and adults aged 12 years and older with HoFH in June 2021.

“This approval heralds a new era for treatment of children 5 to 11 years of age with HoFH,” stated Albert Wiegman, M.D., Ph.D. and Professor, Department of Paediatrics at Amsterdam University Medical Center. “The addition of Evkeeza to aggressive baseline lipid-lowering therapy nearly halves LDL-C levels in a disorder with an extremely high risk of premature cardiovascular disease in young patients, where achieving LDL-C goals otherwise isn’t possible".

Condition: Homozygous Familial Hypercholesterolemia
Type: drug

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