Ultragenyx Pharmaceutical Inc. (RARE) a biopharmaceutical company, on Monday announced that the European Commission has approved Evkeeza for expanded use in treating pediatric patients aged 6 months and older with homozygous familial hypercholesterolemia.
This makes Evkeeza the first approved treatment for young children with this rare, inherited condition that leads to dangerously high cholesterol levels.
Evkeeza (evinacumab) is an angiopoietin-like 3 (ANGPTL3) inhibitor that works by blocking the protein ANGPTL3, which plays a key role in controlling cholesterol levels.
By inhibiting ANGPTL3, Evkeeza reduces LDL-C (bad cholesterol) levels in the blood, helping to manage HoFH, a condition that leads to premature cardiovascular disease. The drug is administered via an infusion every four weeks.
The approval follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in November 2024 and is based on model-based predictions showing significant LDL-C reductions in young patients.
Ultragenyx licensed the ex-US rights to Evkeeza from Regeneron Pharmaceuticals Inc. (REGN) under an agreement signed in January 2022.
Evkeeza was initially approved in 2021 in the European Union as an adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH).
The drug is also approved in the U.S. as an adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 5 years and older, with homozygous familial hypercholesterolemia (HoFH).
"We are excited to offer this life-changing treatment to children with HoFH, providing early intervention to manage LDL-C levels and reduce future cardiovascular risks," said Jane Cooper, Senior Vice President at Ultragenyx.
Cash position:
The company had cash, cash equivalents, and marketable debt securities were $825 million as of September 30, 2024.
RARE shares debuted on the Nasdaq global market on January 31, 2014, under the ticker symbol "RARE", priced at $21.00 per share.
RARE, closed Friday's (Jan. 03, 2025) trading at $41.50, up 0.85%. In Premarket trading, the stock is down 1.08% at $41.50
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