NICE Recommends Ultragenyx’s HoFH Drug

Rare Daily Staff

The National Institute for Health and Care Excellence, the United Kingdom’s drug price watchdog, has issued a final draft guidance recommending Ultragenyx’s Evkeeza to NHS England for a rare lipid disorder.

The recommendation is for Evkeeza 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.

Homozygous familial hypercholesterolemia (HoFH) is a form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and approximately 1,600 people in the European Union. HoFH occurs when two copies of the familial hypercholesterolemia-causing genes are inherited, one from each parent, resulting in dangerously high levels (greater than 400 mg/dL) of LDL-C, or bad cholesterol. Patients with HoFH are at risk of premature atherosclerotic disease and cardiac events at an early age.

Evinacumab, the active substance in Evkeeza, binds to the protein ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every four weeks. It is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.

Regeneron Pharmaceuticals discovered and developed Evkeeza and commercializes the product in HoFH in the United States. Ultragenyx is responsible for commercialization efforts for Evkeeza in HoFH in countries outside of the United States.

NICE determined that, despite uncertainties in the cost-effectiveness evidence comparing Evkeeza with lomitapide (marketed as Juxtapid and Lojuxta) in adults with HoFH, it provided cost savings compared with lomitapide. NICE said that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies, can lower LDL-C levels when statins and other therapies have not reduced them enough.

NICE also acknowledged that adolescent patients with HoFH on lipid-lowering therapies with or without lipoprotein apheresis have a high unmet need because these drugs 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 every four weeks as an infusion.

“This recognition from NICE on the clinical and economic benefits of Evkeeza is a meaningful milestone for people living with HoFH in England and Wales,” said Erik Harris, chief commercial officer at Ultragenyx.

Evkeeza is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., England and Wales, Canada, Italy, and Germany.