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Homozygous Familial Hypercholesterolemia

7
Pipeline Programs
11
Companies
17
Clinical Trials
2 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
1
4
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
583%
RNA Therapeutic
117%
+ 9 programs with unclassified modality

On Market (1)

Approved therapies currently available

Regeneron
EVKEEZAApproved
evinacumab
Regeneron
Angiopoietin-like 3 Inhibitor [EPC]injection2021
7M Part D

Competitive Landscape

11 companies ranked by most advanced pipeline stage

Regeneron
RegeneronTARRYTOWN, NY
5 programs
1
REGN1500 250 mg SC/15 mg/kg IV/450 mg SCPHASE_21 trial
AlirocumabPHASE_3Monoclonal Antibody
EVKEEZA(Evinacumab)PHASE_3Monoclonal Antibody2 trials
evinacumabPHASE_3Monoclonal Antibody1 trial
evinacumabPHASE_3Monoclonal Antibody1 trial
Active Trials
NCT02265952Completed9Est. Jul 2018
NCT06500598Approved For Marketing
NCT04233918Completed20Est. May 2023
+2 more trials
Sandoz
SandozAustria - Kundl
1 program
1
Inclisiran Sodium for injectionPhase 3RNA Therapeutic
LIB Therapeutics
LIB TherapeuticsOH - Cincinnati
1 program
1
lerodalcibepPhase 31 trial
Active Trials
NCT04034485Completed65Est. Jan 2023
Amryt Pharma
Amryt PharmaUK - London
1 program
1
lomitapidePhase 34 trials
Active Trials
NCT06832371Active Not Recruiting73Est. Dec 2026
NCT04681170Completed46Est. Jun 2024
NCT02399839Terminated5Est. Dec 2022
+1 more trials
Arrowhead Pharmaceuticals
1 program
1
zodasiranPhase 32 trials
Active Trials
NCT07473843Not Yet Recruiting12Est. May 2029
NCT07037771Recruiting60Est. Aug 2027
Innovent Biologics
Innovent BiologicsChina - Jiangsu
1 program
1
IBI306Phase 2/31 trial
Active Trials
NCT04031742Completed18Est. Dec 2021
Kite Pharma
Kite PharmaCA - El Segundo
1 program
1
Run-In Period: PlaceboPhase 2
HDL Therapeutics
HDL TherapeuticsFL - Vero Beach
1 program
HDL Therapeutics PDS-2 SystemN/A1 trial
Active Trials
NCT03135184Unknown6Est. Aug 2019
Ionis Pharmaceuticals
1 program
AKCEA-ANGPTL3-LRXPHASE_21 trial
Active Trials
NCT03455777Withdrawn0Est. Dec 2018
Gilead Sciences
Gilead SciencesFOSTER CITY, CA
1 program
Run-In Period: PlaceboPHASE_21 trial
Active Trials
NCT02472535Completed13Est. Feb 2016
Ultragenyx Pharmaceutical
1 program
EvinacumabPHASE_3Monoclonal Antibody1 trial
Active Trials
NCT05611528Completed10Est. Mar 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Arrowhead Pharmaceuticalszodasiran
Arrowhead Pharmaceuticalszodasiran
Ultragenyx PharmaceuticalEvinacumab
Amryt Pharmalomitapide
RegeneronEvinacumab
LIB Therapeuticslerodalcibep
Regeneronevinacumab
Regeneronevinacumab
Innovent BiologicsIBI306
Ionis PharmaceuticalsAKCEA-ANGPTL3-LRX
Gilead SciencesRun-In Period: Placebo
RegeneronREGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Amryt Pharmalomitapide
HDL TherapeuticsHDL Therapeutics PDS-2 System
Amryt Pharmalomitapide

Showing 15 of 16 trials with date data

Clinical Trials (17)

Total enrollment: 818 patients across 17 trials

NCT07473843Arrowhead Pharmaceuticalszodasiran

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Start: Mar 2026Est. completion: May 202912 patients
Phase 3Not Yet Recruiting
NCT07037771Arrowhead Pharmaceuticalszodasiran

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

Start: Jun 2025Est. completion: Aug 202760 patients
Phase 3Recruiting
NCT05611528Ultragenyx PharmaceuticalEvinacumab

Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia

Start: Feb 2023Est. completion: Mar 202510 patients
Phase 3Completed
NCT04681170Amryt Pharmalomitapide

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Start: Dec 2020Est. completion: Jun 202446 patients
Phase 3Completed
NCT04233918RegeneronEvinacumab

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Start: Jun 2020Est. completion: May 202320 patients
Phase 3Completed
NCT04034485LIB Therapeuticslerodalcibep

Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Start: Dec 2019Est. completion: Jan 202365 patients
Phase 3Completed
NCT03409744Regeneronevinacumab

Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Start: Mar 2018Est. completion: Apr 2023116 patients
Phase 3Completed
NCT03399786Regeneronevinacumab

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Start: Jan 2018Est. completion: Mar 202065 patients
Phase 3Completed
NCT04031742Innovent BiologicsIBI306

A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia

Start: Sep 2019Est. completion: Dec 202118 patients
Phase 2/3Completed
NCT03455777Ionis PharmaceuticalsAKCEA-ANGPTL3-LRX

Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Start: Apr 2018Est. completion: Dec 20180
Phase 2Withdrawn
NCT02472535Gilead SciencesRun-In Period: Placebo

Study to Evaluate the Effects of MBX-8025 in Patients With HoFH

Start: Apr 2015Est. completion: Feb 201613 patients
Phase 2Completed
NCT02265952RegeneronREGN1500 250 mg SC/15 mg/kg IV/450 mg SC

Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Start: Feb 2015Est. completion: Jul 20189 patients
Phase 2Completed
NCT06500598RegeneronEvinacumab

Compassionate Use of Evinacumab

N/AApproved For Marketing
NCT06832371Amryt Pharmalomitapide

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

Start: Sep 2024Est. completion: Dec 202673 patients
N/AActive Not Recruiting
NCT03135184HDL TherapeuticsHDL Therapeutics PDS-2 System

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

Start: Jan 2018Est. completion: Aug 20196 patients
N/AUnknown
NCT02399839Amryt Pharmalomitapide

Global Lomitapide Pregnancy Exposure Registry

Start: Oct 2014Est. completion: Dec 20225 patients
N/ATerminated
NCT02135705Amryt Pharmalomitapide

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Start: Mar 2014Est. completion: Sep 2028300 patients
N/ARecruiting

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

4 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 818 patients
11 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.