NHS England Approves Evkeeza®: A Lifeline for HoFH Patients

Evkeeza® Gets the Green Light from NHS England

In a significant move for patients suffering from Homozygous Familial Hypercholesterolaemia (HoFH), NHS England has introduced the commissioning of Evkeeza (evinacumab) for eligible individuals aged 12 years and older. This follows a positive assessment from the National Institute for Health and Care Excellence (NICE). With Evkeeza now available for prescription in seven NHS hospital trusts across the region, it represents a breakthrough in the treatment of this rare cholesterol disorder.

Access to Critical Treatment

NHS England’s decision to routinely commission Evkeeza is a game-changer for those battling HoFH, a severe genetic condition leading to extremely high cholesterol levels. Dr. Jaimini Cegla, a leading clinician in this field, expressed gratitude for the acceptance of NICE recommendations. He highlighted how this treatment will aid patients who often experience early-onset cardiovascular issues due to the limitations of traditional therapies.

What Makes Evkeeza Unique?

Evkeeza is the first monoclonal antibody that targets the angiopoietin-like 3 (ANGPTL3) protein, which plays a crucial role in regulating cholesterol levels. By inhibiting this protein, Evkeeza effectively reduces LDL-C levels in patients with HoFH when used with existing lipid-lowering therapies. This innovation holds promise for those with few alternatives when it comes to managing their condition.

Funding and Resource Allocation

David Nestor, Vice President and General Manager for the UK, Ireland, and Nordics at Ultragenyx, remarked on the significance of NICE’s endorsement, emphasizing that clinical and economic benefits were key factors in NHS England's decision. The approval highlights a commitment to employing cost-effective interventions that can yield substantial health benefits for those in need.

Positive Reactions from Advocacy Groups

Healthcare organizations, including HEART UK, have welcomed this development with enthusiasm. Chief Executive Officer Jules Payne expressed delight at the improved access for HoFH patients, underscoring the positive impact of Evkeeza on their long-term health.

Understanding Homozygous Familial Hypercholesterolaemia

HoFH is a rare but serious genetic condition that presents significant health challenges. It affects roughly 1 in 300,000 individuals globally, with about 1,600 cases in the European Union. The condition arises when both parents pass down the genes responsible for familial hypercholesterolemia, leading to dangerously high cholesterol levels that can precipitate early cardiovascular diseases. Awareness and treatment of HoFH are paramount for improving the quality of life of affected individuals.

About Evkeeza and Its Manufacturer

Evkeeza, approved by the UK MHRA, serves as an adjunctive therapy alongside dietary changes and existing cholesterol-lowering treatments for patients aged five and up with HoFH. The drug, which is administered through intravenous infusion, binds to ANGPTL3, effectively regulating cholesterol levels in the body.

Company Profile: Ultragenyx Pharmaceutical Inc.

Ultragenyx is dedicated to treating rare genetic disorders with innovative therapies. Their robust portfolio targets conditions that currently lack effective treatment options. As they expand the availability of Evkeeza in markets outside the U.S., they reaffirm their commitment to addressing high unmet medical needs.

Important Safety Information

Like all medications, Evkeeza comes with potential side effects. Commonly reported symptoms include those associated with the common cold, particularly nasal congestion. For younger patients, fatigue is a notable concern. However, serious allergic reactions may also occur, underscoring the importance of monitoring during treatment.

Frequently Asked Questions

What is Evkeeza used for?

Evkeeza is used for treating Homozygous Familial Hypercholesterolaemia (HoFH) in patients aged 12 years and older.

Who can access Evkeeza?

It is available to eligible individuals in NHS England, as well as children aged 5 to 11 with prior approval.

How does Evkeeza work?

Evkeeza works by inhibiting the protein ANGPTL3, which helps to lower LDL-C levels in the blood.

What should patients be aware of regarding side effects?

Common side effects include symptoms similar to those of a cold, and in children, feelings of fatigue. Allergic reactions are also a serious possibility.

Who manufactures Evkeeza?

Evkeeza is developed by Regeneron Pharmaceuticals, Inc. and marketed by Ultragenyx Pharmaceutical Inc. in regions outside the U.S.

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