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Heterozygous Familial Hypercholesterolemia

5
Pipeline Programs
10
Companies
8
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
5
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
150%
Small Molecule
150%
+ 9 programs with unclassified modality

Competitive Landscape

10 companies ranked by most advanced pipeline stage

Pfizer
PfizerNEW YORK, NY
1 program
1
BococizumabPhase 3Monoclonal Antibody1 trial
Active Trials
NCT01968980Completed370Est. Apr 2016
Sandoz
SandozAustria - Kundl
1 program
1
FluvastatinPhase 3Small Molecule1 trial
Active Trials
NCT00171236Completed84Est. Mar 2005
Innovent Biologics
Innovent BiologicsChina - Jiangsu
1 program
1
IBI306Phase 31 trial
Active Trials
NCT04179669Completed149Est. Jun 2021
LIB Therapeutics
LIB TherapeuticsOH - Cincinnati
1 program
1
lerodalcibepPhase 3
Medpace
MedpaceCINCINNATI, OH
1 program
1
lerodalcibepPhase 31 trial
Active Trials
NCT04797104Completed478Est. Dec 2023
Regeneron
RegeneronTARRYTOWN, NY
2 programs
LMTPHASE_3
PraluentPHASE_4
Amgen
AmgenTHOUSAND OAKS, CA
1 program
Lp(a) and Aortic Valve CalcificationN/A1 trial
Active Trials
NCT02976818Completed173Est. Feb 2020
Madrigal Pharmaceuticals
Madrigal PharmaceuticalsWEST CONSHOHOCKEN, PA
1 program
MGL-3196PHASE_21 trial
Active Trials
NCT03038022Completed116Est. Jan 2018
Sanofi
SanofiPARIS, France
1 program
LMTPHASE_31 trial
Active Trials
NCT01709500Completed249Est. Jan 2015
Ionis Pharmaceuticals
1 program
mipomersen sodiumPHASE_31 trial
Active Trials
NCT00706849Completed124Est. May 2010

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Medpacelerodalcibep
Innovent BiologicsIBI306
PfizerBococizumab
SanofiLMT
Ionis Pharmaceuticalsmipomersen sodium
SandozFluvastatin
Madrigal PharmaceuticalsMGL-3196
AmgenLp(a) and Aortic Valve Calcification

Clinical Trials (8)

Total enrollment: 1,743 patients across 8 trials

NCT04797104Medpacelerodalcibep

Study to Assess the Efficacy and Safety of LIB003 in HeFH Patients on Oral Lipid Therapy Needing Further LDL-C Reduction

Start: Apr 2021Est. completion: Dec 2023478 patients
Phase 3Completed
NCT04179669Innovent BiologicsIBI306

Safety and Efficacy of IBI306 in HeFH Patients

Start: Dec 2019Est. completion: Jun 2021149 patients
Phase 3Completed
NCT01968980PfizerBococizumab

A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia

Start: Oct 2013Est. completion: Apr 2016370 patients
Phase 3Completed
NCT01709500SanofiLMT

Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)

Start: Dec 2012Est. completion: Jan 2015249 patients
Phase 3Completed
NCT00706849Ionis Pharmaceuticalsmipomersen sodium

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

Start: Jul 2008Est. completion: May 2010124 patients
Phase 3Completed
NCT00171236SandozFluvastatin

Efficacy and Safety of Fluvastatin in Children With Heterozygous Familial Hypercholesterolemia

Start: Oct 2001Est. completion: Mar 200584 patients
Phase 3Completed
NCT03038022Madrigal PharmaceuticalsMGL-3196

Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)

Start: Feb 2017Est. completion: Jan 2018116 patients
Phase 2Completed
NCT02976818AmgenLp(a) and Aortic Valve Calcification

Lp(a) and Aortic Valve Calcification

Start: Apr 2017Est. completion: Feb 2020173 patients
N/ACompleted

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

5 late-stage (Phase 3) programs, potential near-term approvals
10 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.