Ultragenyx receives positive recommendation from NICE in U.K. for Evkeeza

Ultragenyx Pharmaceutical announced that the National Institute for Health and Care Excellence, NICE, has issued a final draft guidance recommending Evkeeza to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia. Evkeeza is the first angiopoietin-like 3 inhibitor treatment indicated for this rare and debilitating condition. 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. 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.