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Evinacumab

Phase 3

Homozygous Familial Hypercholesterolemia | Monoclonal antibody | Metabolic |Ultragenyx Pharmaceutical Inc.|Trials Updated: Mar 25, 2025

Evinacumab development status

Highest phase Phase 3
Registered trials 10 across 2 sponsors since May 2017

Evinacumab target and mechanism

Molecular targetANGPTL3
Target classInhibitor
ModalityMonoclonal antibody

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Evinacumab clinical trials

Evinacumab · 1 trial · 1 indication

Phase 3 1
NCT05611528Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
COMPLETED10 Analytics
PHASE3COMPLETED
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Change in lipid profile
Every 4 weeks up to 2 years

Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration

Change in aspartate transaminase (AST) plasma concentration
Every 4 weeks up to 2 years
change in alanine aminotransferase (ALT) plasma concentration
Every 4 weeks up to 2 years

Secondary Endpoints

Lipoprotein (a)
Yearly up to 2 years
Complete blood count
Every 12 weeks up to 2 years
Total bilirubin
Every 12 weeks up to 2 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Evinacumab-treated patientsEXPERIMENTAL -

Interventions

NameTypeDescription
EvinacumabDRUGEvinacumab 15 mg/kg administered intravenously every 4 weeks
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * HoFH patients having been previously or being currently treated with evinacumab or clinical diagnosis of HoFH requiring additional lipid lowering therapy. Exclusion Criteria: * Any new condition or worsening of an existing condition, which in the opinion of the investigator ...

Countries:Canada
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Frequently asked questions about Evinacumab

What is evinacumab used for?

Evinacumab is being developed for the treatment of homozygous familial hypercholesterolemia, a rare inherited metabolic condition. It is a monoclonal antibody studied in a Phase 3 clinical trial in adults with this condition. It is an investigational therapy and is not described as approved.

What does evinacumab target?

Evinacumab targets ANGPTL3, also known as angiopoietin-like protein 3. It is an inhibitor of ANGPTL3, meaning it acts by blocking the activity of this protein. This mechanism underlies its development for lowering cholesterol in homozygous familial hypercholesterolemia.

Who is developing evinacumab?

Evinacumab is being developed by Ultragenyx Pharmaceutical Inc., which trades under the ticker RARE. The company is the sponsor associated with the clinical development of this monoclonal antibody for homozygous familial hypercholesterolemia.

What phase is evinacumab in?

Evinacumab is in Phase 3 development for homozygous familial hypercholesterolemia. One Phase 3 trial has been completed, and no active trials are currently listed. It is an investigational drug and is not described as FDA approved.

What clinical trials is evinacumab in?

Evinacumab has been studied in the Phase 3 trial NCT05611528, titled Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia. The trial enrolled 10 adults in Canada and is completed. It was an uncontrolled study without randomization or double blinding.