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Homozygous Familial Hypercholesterolemia (HoFH)

3
Pipeline Programs
4
Companies
5
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
1
0
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
1100%
+ 4 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Visirna Therapeutics
Visirna TherapeuticsChina - Shanghai
1 program
1
VSA003Phase 31 trial
Active Trials
NCT06712771Not Yet Recruiting45Est. Dec 2027
Eddingpharm
EddingpharmChina - Shanghai
1 program
1
EDP167Phase 21 trial
Active Trials
NCT07489209Recruiting20Est. Mar 2027
Regenxbio
RegenxbioMD - Rockville
3 programs
1
AAV directed hLDLR gene therapyPhase 1/2Gene Therapy1 trial
Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) PatientsN/A1 trial
Single intravenousN/A1 trial
Active Trials
NCT04148001Completed4Est. Apr 2020
NCT04080050Unknown8Est. Sep 2025
NCT02651675Terminated9Est. Nov 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Visirna TherapeuticsVSA003
EddingpharmEDP167
RegenxbioAAV directed hLDLR gene therapy
RegenxbioIdentifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients
RegenxbioSingle intravenous

Clinical Trials (5)

Total enrollment: 86 patients across 5 trials

NCT06712771Visirna TherapeuticsVSA003

A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients

Start: Dec 2024Est. completion: Dec 202745 patients
Phase 3Not Yet Recruiting
NCT07489209EddingpharmEDP167

A Dose-exploration Study of EDP167 in HoFH

Start: Feb 2026Est. completion: Mar 202720 patients
Phase 2Recruiting
NCT02651675RegenxbioAAV directed hLDLR gene therapy

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Start: Mar 2016Est. completion: Nov 20209 patients
Phase 1/2Terminated
NCT04148001RegenxbioIdentifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Start: Dec 2019Est. completion: Apr 20204 patients
N/ACompleted
NCT04080050RegenxbioSingle intravenous

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Start: Sep 2019Est. completion: Sep 20258 patients
N/AUnknown

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 86 patients
4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.