04.01.2024 16:40:28

Ultragenyx Pharma Gets Positive Recommendation From NICE For Evkeeza

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) Thursday announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza (evinacumab) to NHS England.

Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.

"We are very pleased that NICE has approved the use of evinacumab within the NHS. Our patients with HoFH often have early onset heart disease, in many cases in their teens, and many of the usual medicines we use to treat cholesterol have limited effectiveness in this condition," stated Dr. Jaimini Cegla, clinical lead of the Lipid and Cardiovascular Risk Service, Hammersmith Hospital. "Evinacumab, which is effective at lowering LDL-C in HoFH when combined with other lipid-lowering therapies, is a much needed and very welcome addition to help us treat our patients as best we can."

"We are delighted that patients with HoFH will have another tool to help manage their condition, especially as it is approved from age 12," stated Jules Payne, CEO of Heart UK.

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