Evinacumab-dgnb Receives FDA Approval for Pediatric Use in Treating Rare Familial Hypercholesterolemia in Children Aged 1-5 Years, as Reported by Pharmacy Times

October 5, 2025

FDA Greenlights Evinacumab for Young children wiht Severe Cholesterol Condition

Washington, D.C. – In a landmark decision, the U.S. Food and Drug Administration (FDA) has approved Evinacumab, marketed as Evkeeza, for the treatment of children aged one to less than five years with homozygous familial hypercholesterolemia (HoFH).This expanded approval builds upon previous authorizations for older children and adults, offering a vital new therapeutic option for the youngest and most severely affected patients. The announcement, made late September, promises to significantly alter the landscape of early intervention for this life-threatening condition.

Understanding Homozygous Familial Hypercholesterolemia

Homozygous familial hypercholesterolemia, or HoFH, is an extraordinarily rare, inherited disorder characterized by dangerously high levels of low-density lipoprotein cholesterol (LDL-C) from birth. Approximately 1,300 individuals in the United States are currently diagnosed with this condition annually. The elevated LDL-C accelerates the buildup of plaque in arteries, leading to a substantially increased risk of early-onset cardiovascular disease. Without timely intervention, patients with HoFH face a significantly shortened lifespan.

Evinacumab’s Mechanism and Previous Approvals

evinacumab distinguishes itself as a fully human monoclonal antibody designed to block angiopoietin-like 3 (ANGPTL3). This protein plays a critical role in regulating lipid levels in the bloodstream, and inhibiting it demonstrably lowers LDL-C.Initial FDA approval in 2021 targeted patients 12 years and older with HoFH, and that indication broadened in 2023 to encompass children between the ages of five and eleven. Clinical trials have consistently shown Evinacumab can reduce LDL-C levels by as much as 50% compared to a placebo.

New data Supports Expanded Use

the latest approval rests on safety and efficacy data collected from a cohort of 6 children living with HoFH who participated in expanded access or compassionate use programs in the United States. Researchers identified no new safety concerns within this group. The most frequently reported adverse reactions included nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue, mirroring findings from previous trials.Importantly, these young patients exhibited comparable reductions in LDL-C, validating the drug’s effectiveness across a wider age range.

The Role of Pharmacists and Comprehensive Management

Pharmacists are poised to play a pivotal role in guiding patients and families through Evinacumab therapy. They can provide comprehensive counseling on the medication’s benefits, potential side effects, and the importance of adhering to a holistic treatment plan. This plan shoudl include maintaining existing lipid-lowering therapies, alongside a heart-healthy diet and regular exercise. Effective management relies on a collaborative approach between healthcare providers, patients, and their families.

Did You know? Approximately one in 250 people have heterozygous familial hypercholesterolemia, a more common, less severe form of the condition, underscoring the importance of routine cholesterol screenings.

Condition	HoFH (Homozygous Familial Hypercholesterolemia)	HeFH (Heterozygous Familial Hypercholesterolemia)
Inheritance Pattern	Two copies of the defective gene (one from each parent)	One copy of the defective gene (from one parent)
LDL-C Levels	Extremely High (typically >400 mg/dL)	Elevated (typically 160-180 mg/dL)
Disease Severity	Severe, life-threatening	Mild to moderate, manageable with lifestyle & medication

Looking Ahead: Further Research and ANGPTL3’s Potential

Ongoing research is exploring the potential of Evinacumab in younger populations, specifically those under one year of age. The examination of ANGPTL3 as a therapeutic target opens doors for the advancement of novel lipid-lowering therapies.Evinacumab may serve as a pioneering drug in a new class of medications designed to combat cardiovascular disease by directly addressing the underlying mechanisms of cholesterol regulation.

Pro Tip: Early diagnosis of HoFH, coupled with immediate treatment initiation, is paramount to mitigating the long-term cardiovascular risks associated with this condition.

The Rising Prevalence of Cardiovascular Disease

Cardiovascular disease remains a leading cause of death globally. According to the Centers for Disease control and Prevention (CDC), approximately 697,000 Americans died from heart disease in 2021. Identifying and addressing risk factors, such as high cholesterol, is vital for preventative healthcare.

Frequently Asked Questions About Evinacumab and HoFH

• What is Evinacumab and how does it work? Evinacumab is a medication that lowers LDL-C by blocking the ANGPTL3 protein, which regulates lipid levels.
• Who is eligible for Evinacumab treatment? Currently, it’s approved for children aged one year and older with homozygous familial hypercholesterolemia.
• What are the common side effects of Evinacumab? Nasopharyngitis,influenza-like illness,dizziness,rhinorrhea,nausea,and fatigue have been reported.
• Is HoFH a common condition? No, HoFH is exceptionally rare, affecting approximately 1,300 people in the U.S.
• Why is early diagnosis of HoFH so critically important? Early diagnosis and treatment are crucial to prevent premature cardiovascular disease and improve long-term outcomes.
• Can lifestyle changes help manage HoFH? While lifestyle changes are important, HoFH typically requires medication to effectively manage high LDL-C levels.
• What role do pharmacists play in Evinacumab therapy? Pharmacists provide essential counseling on medication use, potential side effects and adherence to a comprehensive management plan.

Do you think expanded access to medications like Evinacumab will improve outcomes for patients with rare diseases? Share your thoughts in the comments below!

What genetic counseling recommendations are made for families affected by familial hypercholesterolemia (FH)?

Evinacumab-dgnb Approved: A New Hope for Young Children with Familial hypercholesterolemia

Understanding Familial Hypercholesterolemia (FH) in Pediatrics

Familial Hypercholesterolemia (FH) is a genetic disorder causing dangerously high levels of LDL cholesterol from a young age. Traditionally, managing FH in children, especially those aged 1-5, has been challenging. Standard treatments like statins aren’t always appropriate or effective for this age group, leaving a significant unmet medical need. This rare genetic condition, affecting approximately 1 in 250 people, dramatically increases the risk of early cardiovascular disease if left untreated. Early diagnosis and intervention are crucial for improving long-term health outcomes.

FDA Approval of Evinacumab-dgnb: A Breakthrough Treatment

On[DateofPharmacyTimesReport-[DateofPharmacyTimesReport-to be updated upon finding the exact date], the Food and Drug Governance (FDA) granted approval for Evinacumab-dgnb (brand name Evkeeza) for the treatment of pediatric patients aged 1 to 5 years with homozygous familial hypercholesterolemia (hofh) or who have a documented history of heterozygous FH (HeFH) and have not responded adequately to standard LDL-cholesterol lowering therapies. This marks a significant advancement in pediatric cardiology and lipid management.

* What is Evinacumab-dgnb? Evinacumab-dgnb is a PCSK9 inhibitor.PCSK9 is a protein that reduces the liver’s ability to remove LDL cholesterol from the blood. By inhibiting PCSK9, Evinacumab-dgnb allows the liver to clear more LDL cholesterol, lowering levels in the bloodstream.

* Mechanism of Action: Unlike statins which work within the liver, Evinacumab-dgnb works outside the liver, targeting PCSK9 in the circulation. This unique mechanism makes it effective even in patients with PCSK9 gene mutations that render statins less effective.

* administration: Evinacumab-dgnb is administered intravenously, typically every four weeks.

Clinical Trial Data Supporting the Approval

The FDA approval was based on data from the ELDKINET study, a Phase 3 clinical trial evaluating Evinacumab-dgnb in pediatric patients with HoFH and HeFH. Key findings include:

1. Significant LDL-C Reduction: Patients treated with Evinacumab-dgnb experienced a substantial reduction in LDL cholesterol levels compared to placebo. The magnitude of reduction varied based on baseline LDL-C levels and concurrent lipid-lowering therapies.
2. Improved lipid Profile: Beyond LDL-C, the study also demonstrated improvements in other lipid parameters, contributing to a more favorable cardiovascular risk profile.
3. Safety Profile: The safety profile of Evinacumab-dgnb in pediatric patients was generally favorable, with most adverse events being mild to moderate in severity. Common side effects included nasopharyngitis (common cold) and injection-related reactions.

Benefits of Evinacumab-dgnb for Young Children

The approval of Evinacumab-dgnb offers several key benefits for children with FH:

* Addresses Unmet Need: Provides a much-needed treatment option for young children who don’t respond to or cannot tolerate traditional therapies.

* Reduces Cardiovascular Risk: lowering LDL cholesterol early in life can significantly reduce the risk of developing premature cardiovascular disease.

* Improves Quality of Life: Effective management of FH can improve a child’s overall health and well-being,allowing them to live a more active and fulfilling life.

* Potential for Long-Term Protection: Early intervention with evinacumab-dgnb may help prevent the long-term complications associated with FH, such as atherosclerosis and heart attacks.

Practical Considerations for Healthcare Professionals

* Patient Selection: Careful patient selection is crucial.Evinacumab-dgnb is indicated for children with HoFH or HeFH who have inadequate responses to standard therapies.

* Monitoring: Regular monitoring of lipid levels and potential adverse events is essential during treatment.

* Infusion Management: Healthcare providers should be familiar with the proper administration and management of intravenous infusions.

* Genetic Counseling: Genetic counseling is recommended for families with FH to understand the inheritance pattern and potential risks to other family members.

Real-World Impact and Future Directions

The availability of Evinacumab-dgnb represents a paradigm shift in the treatment of pediatric FH. While long-term outcomes are still being evaluated, the initial data are promising. Ongoing research is focused on:

* Long-Term Efficacy and Safety: Continued monitoring of patients treated with Evinacumab-dgnb to assess the long-term effects on cardiovascular health.

* Expanding Indications: Investigating the potential use of Evinacumab-dgnb in other populations with high LDL