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Familial Hypercholesterolemia

3
Pipeline Programs
13
Companies
6
Clinical Trials
2 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
0
1
0
1
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 14 programs with unclassified modality

Competitive Landscape

12 companies ranked by most advanced pipeline stage

B. Braun
B. BraunGermany - Melsungen
1 program
1
HELP SecuraPhase 41 trial
Active Trials
NCT00916643Completed113Est. Sep 2009
Viatris
ViatrisCANONSBURG, PA
1 program
1
atorvastatinPhase 3Small Molecule1 trial
Active Trials
NCT00827606Completed272Est. Oct 2013
Sandoz
SandozAustria - Kundl
1 program
1
APL180Phase 2
Pfizer
PfizerNEW YORK, NY
2 programs
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
Russian Familial Hypercholesterolemia RegistryN/A
Amgen
AmgenTHOUSAND OAKS, CA
2 programs
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A1 trial
Russian Familial Hypercholesterolemia RegistryN/A
Active Trials
NCT02009345Recruiting6,000Est. Nov 2028
Genome & Company
Genome & CompanyKorea - Suwon
2 programs
Research-based genetic test for Familial HypercholesterolemiaN/A1 trial
Return of Actionable Variants Empirical StudyN/A
Active Trials
NCT05218005Completed140Est. Aug 2025
Sanofi
SanofiPARIS, France
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
MSD
MSDIreland - Ballydine
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
Merck & Co.
Merck & Co.RAHWAY, NJ
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
Sharp Therapeutics
Sharp TherapeuticsPA - Pittsburgh
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
Return of Actionable Variants Empirical StudyN/A1 trial
Active Trials
NCT03640234Completed418Est. Sep 2022
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
Russian Familial Hypercholesterolemia RegistryN/A1 trial
Active Trials
NCT02208869Recruiting1,000Est. Dec 2026

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
B. BraunHELP Secura
Viatrisatorvastatin
Genome & CompanyResearch-based genetic test for Familial Hypercholesterolemia
Human BioSciencesReturn of Actionable Variants Empirical Study
AstraZenecaRussian Familial Hypercholesterolemia Registry
AmgenFamilial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Clinical Trials (6)

Total enrollment: 7,943 patients across 6 trials

NCT00916643B. BraunHELP Secura

Low-Density Lipoprotein (LDL) Apheresis Using H.E.L.P. Therapy

Start: Dec 1999Est. completion: Sep 2009113 patients
Phase 4Completed
NCT00827606Viatrisatorvastatin

Atorvastatin Three Year Pediatric Study

Start: Mar 2009Est. completion: Oct 2013272 patients
Phase 3Completed
NCT05218005Genome & CompanyResearch-based genetic test for Familial Hypercholesterolemia

Advancing Cardiac Care Unit-based Rapid Assessment and Treatment of hypErcholesterolemia

Start: Jan 2022Est. completion: Aug 2025140 patients
N/ACompleted
NCT03640234Human BioSciencesReturn of Actionable Variants Empirical Study

Return of Actionable Variants Empirical Study

Start: Feb 2016Est. completion: Sep 2022418 patients
N/ACompleted
NCT02208869AstraZenecaRussian Familial Hypercholesterolemia Registry

Russian Familial Hypercholesterolemia Registry

Start: Jan 2014Est. completion: Dec 20261,000 patients
N/ARecruiting
NCT02009345AmgenFamilial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Start: Nov 2013Est. completion: Nov 20286,000 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

1 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 7,943 patients
13 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.