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Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)
NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England.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.” NICE deemed that, despite uncertainties in the cost-effectiveness evidence comparing evinacumab with lomitapide in adults with HoFH, overall there are cost savings with evinacumab compared with lomitapide. The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough. NICE also acknowledged that adolescent patients with HoFH on LLTs with or without lipoprotein apheresis have a high unmet need because LLTs have limited effectiveness in this patient population, lipoprotein apheresis can be traumatic and time consuming, and there is potential for an inequality of access if made available to adults and not adolescents. NICE considered that Evkeeza addresses an unmet need for new treatments to lower LDL-C, and that Evkeeza may improve adherence to treatment because it is administered once monthly (every 4 weeks) as an infusion. “We are delighted that patients with HoFH will have another tool to help manage their condition, especially as it is approved ...