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Familial Hypercholesterolemia - Heterozygous

4
Pipeline Programs
4
Companies
4
Clinical Trials
1 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
3
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
3100%
+ 2 programs with unclassified modality

On Market (1)

Approved therapies currently available

Novartis
LEQVIOApproved
inclisiran
Novartis
subcutaneous2021
11M Part D

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Novartis
NovartisBASEL, Switzerland
1 program
1
LEQVIO(Inclisiran)PHASE_3RNA Therapeutic
Sandoz
SandozAustria - Kundl
2 programs
2
InclisiranPhase 3RNA Therapeutic1 trial
InclisiranPhase 3RNA Therapeutic1 trial
Active Trials
NCT04652726Completed141Est. Nov 2024
NCT06597019Recruiting51Est. Apr 2029
LIB Therapeutics
LIB TherapeuticsOH - Cincinnati
1 program
1
lerodalcibep 300 mg Monthly SCPhase 31 trial
Active Trials
NCT07102511Not Yet Recruiting150Est. Jun 2027
Amgen
AmgenTHOUSAND OAKS, CA
1 program
MYOCARDIAL SILENT INFARCTIONS AND FIBROSIS IN FAMILIAL HYPERCHOLESTEROLEMIA (CHOLCOEUR)N/A1 trial
Active Trials
NCT02517944Unknown110Est. Nov 2015

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
LIB Therapeuticslerodalcibep 300 mg Monthly SC
SandozInclisiran
SandozInclisiran
AmgenMYOCARDIAL SILENT INFARCTIONS AND FIBROSIS IN FAMILIAL HYPERCHOLESTEROLEMIA (CHOLCOEUR)

Clinical Trials (4)

Total enrollment: 452 patients across 4 trials

NCT07102511LIB Therapeuticslerodalcibep 300 mg Monthly SC

Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH

Start: Sep 2025Est. completion: Jun 2027150 patients
Phase 3Not Yet Recruiting
NCT06597019SandozInclisiran

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

Start: Dec 2024Est. completion: Apr 202951 patients
Phase 3Recruiting
NCT04652726SandozInclisiran

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

Start: Jan 2021Est. completion: Nov 2024141 patients
Phase 3Completed
NCT02517944AmgenMYOCARDIAL SILENT INFARCTIONS AND FIBROSIS IN FAMILIAL HYPERCHOLESTEROLEMIA (CHOLCOEUR)

MYOCARDIAL SILENT INFARCTIONS AND FIBROSIS IN FAMILIAL HYPERCHOLESTEROLEMIA (CHOLCOEUR)

Start: Nov 2014Est. completion: Nov 2015110 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

3 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 452 patients
RNA Therapeutic is the dominant modality (100% of programs)
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.