NHS England Introduces Evkeeza for HoFH Patients Aged 12+

LONDON, Dec. 20, 2024 – Ultragenyx Pharmaceutical Inc., a company renowned for its focus on rare genetic diseases, has announced a significant development concerning its product, Evkeeza (evinacumab). Following guidance from the National Institute for Health and Care Excellence (NICE) in September, NHS England has now commenced the commissioning of Evkeeza. This move will allow patients aged 12 and older within specific criteria to access this treatment across seven hospital trusts in England. Moreover, provisions have been made for children aged 5 to 11 through the NHS England Commissioning Medicines for Children policy.

Dr. Jaimini Cegla, who leads the Lipid and Cardiovascular Risk Service at Hammersmith Hospital, expressed satisfaction with this progress. She highlighted the challenges faced by patients with Homozygous Familial Hypercholesterolemia (HoFH), who often experience heart disease early in life. Standard cholesterol treatments frequently prove ineffective for these individuals. Evinacumab, by effectively lowering LDL-C when used with other lipid-lowering therapies, presents a promising new option for treating HoFH.

Eligible individuals with HoFH in England, Wales, and Northern Ireland will now have access to Evkeeza, marking the first instance of a commercialized monoclonal antibody that inhibits the angiopoietin-like 3 protein (ANGPTL3). The commissioning of Evkeeza will occur at seven designated NHS Trusts, including Bristol Teaching Hospital NHS Trust and University Hospital Birmingham.

David Nestor, Ultragenyx's Vice President and General Manager for the UK, Ireland, and Nordics, acknowledged the role of NICE in highlighting both the clinical and economic benefits of Evkeeza. He praised the recognition of the therapy as a cost-effective use of NHS resources, which facilitated this advancement. Nestor also expressed gratitude to both the medical and patient communities for their support in securing access to Evkeeza for those in need.

Jules Payne, CEO of HEART UK, shared enthusiasm for the treatment's availability, noting the positive impact it will have on patients living with HoFH.

Homozygous Familial Hypercholesterolemia (HoFH) is a severe inherited condition that affects approximately 1 in 300,000 people worldwide, with around 1,600 cases within the European Union. The condition results from inheriting two copies of genes that cause familial hypercholesterolemia, leading to dangerously high LDL-C levels. Individuals with HoFH are at a heightened risk of early-onset atherosclerotic disease and cardiac events.

Evkeeza has received approval from the UK MHRA for use alongside diet and other LDL-C lowering treatments in patients aged 5 years and older with HoFH. Evinacumab, the active ingredient, targets and blocks ANGPTL3, a protein that regulates cholesterol. By inhibiting ANGPTL3, Evkeeza effectively lowers blood cholesterol levels and is administered intravenously.

Regeneron Pharmaceuticals, Inc. initially discovered and developed Evkeeza and markets it in the U.S. under the name evinacumab-dgnb. Outside the U.S., Ultragenyx manages the commercialization of Evkeeza for HoFH.

While Evkeeza offers promising benefits, it is important to be aware of potential side effects. Common reactions include symptoms similar to a cold, such as a runny nose, and fatigue in children aged 5 to 11. Serious allergic reactions are also possible, and comprehensive product information should be consulted.

Ultragenyx Pharmaceutical Inc. is committed to providing innovative solutions for rare genetic diseases. The company's product portfolio is designed to address conditions with significant unmet needs, focusing on diseases with clear biological pathways where treatment options are limited.