Familial Hypercholesterolemia

2
Pipeline Programs
6
Companies
3
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
0
0
0
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
1100%
+ 7 programs with unclassified modality

On Market (1)

Approved therapies currently available

Amgen
REPATHAApproved
evolocumab
Amgen
PCSK9 Inhibitor [EPC]injection2015

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Amgen
AmgenTHOUSAND OAKS, CA
3 programs
1
EvolocumabPhase 4Monoclonal Antibody1 trial
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A1 trial
Russian Familial Hypercholesterolemia RegistryN/A1 trial
Active Trials
NCT02009345Recruiting6,000Est. Nov 2028
NCT02208869Recruiting1,000Est. Dec 2026
NCT03331666Terminated4Est. Feb 2020
Pfizer
PfizerNEW YORK, NY
2 programs
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
Russian Familial Hypercholesterolemia RegistryN/A
Sanofi
SanofiPARIS, France
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
M&
Merck & Co.RAHWAY, NJ
1 program
Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale CanadaN/A
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
Russian Familial Hypercholesterolemia RegistryN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
AmgenEvolocumab
AmgenRussian Familial Hypercholesterolemia Registry
AmgenFamilial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Clinical Trials (3)

Total enrollment: 7,004 patients across 3 trials

Impact of LDL-cholesterol Lowering on Platelet Activation

Start: Nov 2018Est. completion: Feb 20204 patients
Phase 4Terminated
NCT02208869AmgenRussian 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

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 actively recruiting trials targeting 7,004 patients
6 companies competing in this space