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Ultragenyx’s Evkeeza® Secures EC Approval for Young Children Battling Rare Cholesterol Condition
Ultragenyx has received EC approval for Evkeeza® to treat children with homozygous familial hypercholesterolemia from 6 months old.
Expansion of Treatment Options for Rare Disease
Ultragenyx Pharmaceutical Inc. announced that the European Commission has expanded the approval of Evkeeza® (evinacumab) for use in children aged 6 months and older with homozygous familial hypercholesterolemia (HoFH). This drug is now the first approved treatment for very young children in the EU with this condition, which leads to dangerously high levels of low-density lipoprotein cholesterol (LDL-C). This decision followed a positive recommendation from the Committee for Medicinal Products for Human Use in November 2024. Clinical research supports Evkeeza’s effectiveness in reducing LDL-C levels among this younger group. Global support for this approval has been strong, with Evkeeza now available in countries including the UK, U.S., Canada, and several EU nations.
Advantages of Approval
- The European Commission’s approval allows Evkeeza® to treat children from 6 months, a critical step in pediatric healthcare.
- It is notable as the first medicine approved in the EU for very young patients with HoFH, underlining Ultragenyx’s commitment to children with rare diseases.
- The positive recommendation indicates regulatory confidence in Evkeeza’s safety and effectiveness for younger users.
- Expanding Evkeeza’s indications increases the market potential and strengthens Ultragenyx’s position in rare disease therapeutics.
Concerns Over Treatment
- Evidence for Evkeeza’s effectiveness in children under 5 is based on model predictions, raising questions about the strength of the data.
- Though effective in compassionate use for a small group, there is concern that small sample sizes might not reflect broader pediatric outcomes.
- There are safety concerns regarding drug administration to young children, especially since its use is not recommended for those under 6 months due to lack of data.
Frequently Asked Questions
What is Evkeeza and its approved age for treatment?
Evkeeza (evinacumab) is approved for children aged 6 months and older with homozygous familial hypercholesterolemia (HoFH) in the EU.
How does Evkeeza work to lower cholesterol?
Evkeeza is an ANGPTL3 inhibitor that reduces cholesterol levels by blocking the protein responsible for cholesterol regulation in the blood.
What are the side effects of Evkeeza?
Common side effects may include cold-like symptoms such as a runny nose and fatigue in children under 11, and serious allergic reactions can also occur.
Why is the approval of Evkeeza significant for children with HoFH?
The approval offers a vital treatment option for very young children at risk of severe cardiovascular issues due to high LDL-C levels.
What is homozygous familial hypercholesterolemia (HoFH)?
HoFH is a rare genetic disorder that results in extremely high levels of low-density lipoprotein cholesterol, increasing the risk of early cardiovascular disease.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
BASEL, Switzerland, Jan. 06, 2025 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, today announced that the European Commission (EC) has extended the approval of Evkeeza
®
(evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 6 months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor, is the first medicine indicated for children in the European Union (EU) as young as 6 months to treat HoFH, a disease associated with dangerously high levels of low-density lipoprotein cholesterol (LDL-C).
“For very young children suffering from homozygous HoFH, treatment options are limited, and many do not reach treatment goals, leaving them with an uncertain future,” stated Albert Wiegman, M.D., Ph.D., from Amsterdam University Medical Center. “Evinacumab has shown major LDL-C reductions in older children and adolescents. The current expansion for those younger illustrates its potential to aid even earlier in their disease course.”
This EC decision comes after the positive recommendation received from the Committee for Medicinal Products for Human Use in November 2024. The drug’s efficacy for children aged 6 months to under 5 years has been predicted using model-based extrapolation. Analyses indicate that these young patients can expect similar or greater LDL-C reduction after 24 weeks on a 15 mg/kg dose every 4 weeks compared to adults. Additionally, supportive data from five patients further bolsters the case for Evkeeza’s use in this age group.
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Evkeeza Approved for Young Patients with HoFH: A Breakthrough in Cholesterol Management
Substantial LDL-C Reductions Observed in Children Under 5
Children aged between 1 and 4 years who have Homozygous Familial Hypercholesterolemia (HoFH) and received Evkeeza through compassionate use demonstrated a notable decrease in LDL-C levels. This reduction aligns closely with those observed in older patients participating in clinical trials. Currently available data suggests that the safety profile for pediatric patients aged 6 months to 5 years will likely mirror that of older youths. No new safety issues have emerged from the compassionate use program.
Community Celebration and Call for Broader Access
Magdalena Daccord, CEO of the FH Europe Foundation, expressed the international community’s support for this approval, calling it a potentially transformative treatment for families grappling with this serious condition. She emphasized the importance of combining such innovative treatments with early screening and lifestyle management. Daccord stated, “Success will be measured by the availability of this therapy to all those who need it promptly.”
From Approval to Accessibility
Evkeeza was first approved by the European Commission (EC) in June 2021 as a supplementary option for managing cholesterol in adults and adolescents aged 12 and older with HoFH. In December 2023, this approval was expanded to include children aged 5 to 11. The treatment is now covered and available for prescription in several countries, including the UK, U.S., Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg, with early access available in 13 other nations such as Austria and France.
Understanding the Urgency of HoFH
HoFH is a severe genetic condition affecting approximately 1 in 300,000 people worldwide. This illness results from mutations in cholesterol-regulating genes inherited from both parents, leading to very high levels of LDL-C (over 400 mg/dL). Without prompt treatment, individuals with HoFH are at an increased risk for early heart disease.
An Overview of Evkeeza
Evkeeza, with the active ingredient evinacumab, works by inhibiting a protein called ANGPTL3, which is crucial in cholesterol management. This monthly infusion is the first approved therapy aimed specifically at lowering LDL-C levels in children as young as 6 months.
Regulatory and Corporate Overview
Regeneron Pharmaceuticals, Inc. discovered Evkeeza, which is marketed as evinacumab-dgnb in the U.S. Ultragenyx handles the product’s development in markets outside the United States.
Important Safety Information
Patients using Evkeeza may experience common side effects such as cold symptoms and fatigue, especially in children under 11 years. Serious allergic reactions can occur, requiring immediate medical attention. Evkeeza is not recommended for children under 6 months due to insufficient safety data.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical firm dedicated to developing treatments for rare genetic diseases. The company’s strategic focus is to expedite drug development while ensuring patient safety and efficacy.
You can learn more about Ultragenyx by visiting www.ultragenyx.com.
Forward-Looking Statements Disclaimer
This announcement contains forward-looking statements as defined under the Private Securities Litigation Reform Act, including projections about future clinical programs and regulatory plans.
Ultragenyx Faces Potential Challenges in Clinical Development and Market Performance
Understanding the risks and uncertainties ahead for Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc. emphasizes that its future performance hinges on numerous factors, as outlined in the Litigation Reform Act of 1995. This includes its clinical development initiatives, market success of products, collaborations, and overall financial results. However, several risks can lead to significant deviations from the company’s estimates.
Potential challenges include:
- Dependence on third-party partners for critical activities.
- Possible termination of collaboration agreements, such as the one with Regeneron for Evkeeza’s commercialization outside the U.S.
- Delays and uncertainties in clinical drug development and regulatory approval processes.
- Smaller-than-expected market opportunities for their products.
- Variability in distributor and specialty pharmacy behaviors.
- Intense competition impacting product performance.
- Risks of serious side effects stemming from product usage.
- Challenges in scaling the commercial organization.
- Market acceptance issues for current and future offerings.
- Insurance coverage and reimbursement uncertainties.
- Manufacturing hurdles and supply chain disruptions.
- Competition from alternative therapies.
- Concerns about cash sufficiency to support operations and clinical trial timelines.
Ultragenyx clarifies that it does not assume responsibility for updating its forward-looking statements, acknowledging the volatile nature of the pharmaceutical landscape.
For a detailed view of the risks and uncertainties that could impact Ultragenyx, investors are encouraged to review the company’s Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on November 6, 2024, along with other periodic reports.
Besides SEC filings, Ultragenyx reinforces its commitment to transparency through press releases, public conference calls, and various online platforms. Investors can find crucial information on Ultragenyx’s Investor Relations website (
https://ir.ultragenyx.com/
) and LinkedIn (
https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/
).
Contacts
Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
+1-415-475-6370
ir@ultragenyx.com
Media
Joey Fleury
+1-925-784-5829
media@ultragenyx.com
This article was originally published on Quiver News; read the full story for further details.
The views and opinions expressed herein are those of the author and do not necessarily reflect those of Nasdaq, Inc.
