EC expands use of Evkeeza for pediatric patients with HoFH

19 December 2023

The European Commission has extended the approval of Evkeeza (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged five to 11 years with homozygous familial hypercholesterolemia (HoFH).

The drug is marketed in Europe by the USA’s Ultragenyx Pharmaceuticals (Nasdaq: RARE), which gained ex-USA rights to Evkeeza from Regeneron (Nasdaq: REGN) in 2022, for a $30 million upfront payment and committing to pay up to $63 million in additional potential regulatory and sales milestone.

HoFH is a serious, ultra-rare disease that is inherited from one's parents that makes the body unable to clear bad cholesterol. Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as five years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH.

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