Evkeeza® Now Available for Young Children with HoFH

European Commission Approves Evkeeza for Young Patients

BASEL, Switzerland — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a leader in developing treatments for rare and ultrarare genetic diseases, has made significant strides in pediatric care. The European Commission has now approved Evkeeza® (evinacumab) for children as young as 6 months, marking a historic moment for treating homozygous familial hypercholesterolemia (HoFH).

Impact of HoFH on Young Children

Homozygous familial hypercholesterolemia is a rare genetic disorder that results in dangerously high levels of low-density lipoprotein cholesterol (LDL-C). This condition can lead to early cardiovascular diseases, making timely intervention crucial. With limited treatment options available for very young children suffering from HoFH, this new approval provides a hope that many families have eagerly awaited.

Significance of the Approval

Albert Wiegman, M.D., Ph.D., from the Department of Pediatrics at Amsterdam University Medical Center, shared his insights on the approval, emphasizing that it paves the way for treating young children effectively. Previously, treatment options were scarce, and many children struggled to achieve healthy cholesterol levels, which is vital for their long-term health.

Clinical Efficacy and Safety of Evkeeza

The decision by the European Commission comes after a positive recommendation from the Committee for Medicinal Products for Human Use. The scientific data suggest that pediatric patients aged 6 months to under 5 years, receiving Evkeeza, are likely to see significant reductions in LDL-C levels comparable to older patients as they continue with a low-fat diet and other cholesterol-lowering therapies.

Compassionate Use Insights

Supportive data from five patients aged between 1 and 4 years who participated in a compassionate use program also indicated a meaningful reduction in LDL-C levels. These results offer a promising outlook for broader treatment applications in younger age groups.

Global Access to Evkeeza

Evkeeza was first approved for adults and adolescents aged 12 years and older in June 2021. Following the recent expansion of indications to include children aged 5 to 11, it is now officially accessible for all patients aged 6 months and older in the European Union. The treatment is being reimbursed in multiple regions, including the UK, U.S., Canada, Japan, and several EU countries. Early access schemes in additional countries further enhance its availability.

Community Response

Magdalena Daccord, the CEO of the FH Europe Foundation, articulated the sentiments of countless families affected by HoFH, stating the profound impact the approval will have on improving life quality for these children. Advocates are committed to increasing awareness and early diagnosis to ensure young patients can benefit from innovative therapies like Evkeeza.

Understanding Evinacumab

Evinacumab, the active ingredient in Evkeeza, functions as an ANGPTL3 inhibitor. By blocking this protein's effects, Evkeeza plays a crucial role in reducing LDL-C levels. Administered via infusion every month, it provides a vital tool for managing cholesterol levels effectively in young patients.

Safety Measures and Recommendations

While Evkeeza shows great promise, it is crucial to be informed about the potential side effects, which can include common cold symptoms and, in some cases, serious allergic reactions. Parents and caregivers are advised to monitor their children closely and consult healthcare providers regarding any concerns.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is dedicated to developing innovative therapies for serious genetic diseases. Focused on addressing high unmet medical needs, their commitment to swift and responsible drug development continues to yield therapies that enhance the quality of life for affected individuals.

Frequently Asked Questions

What is Evkeeza used for?

Evkeeza is used to treat homozygous familial hypercholesterolemia in children aged 6 months and older.

What are the common side effects of Evkeeza?

The most common side effects include symptoms of the common cold, such as runny nose and fatigue in younger children.

How does Evkeeza work?

Evinacumab, the active substance in Evkeeza, blocks ANGPTL3, a protein involved in regulating cholesterol, effectively lowering cholesterol levels in the blood.

Who can use Evkeeza?

Evkeeza is recommended for patients aged 6 months and older diagnosed with homozygous familial hypercholesterolemia.

Is Evkeeza widely available?

Yes, Evkeeza is now commercially available in several countries and is also accessible through early access schemes in others.

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