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Evinacumab

Phase 3

Homozygous Familial Hypercholesterolemia | Small molecule | Metabolic |Ultragenyx Pharmaceutical Inc.|Last Updated: Mar 25, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLED
Total Trials1
Total Enrollment10
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05611528Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial HypercholesterolemiaPHASE3 COMPLETED 10Feb 21, 2023Mar 18, 2025Mar 25, 20251 Canada
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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 — 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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