Homozygous Familial Hypercholesterolemia

15
Pipeline Programs
7
Companies
29
Clinical Trials
3 recruiting
3
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
4
1
7
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
6100%
+ 7 programs with unclassified modality

On Market (3)

Approved therapies currently available

Regeneron
EVKEEZAApproved
evinacumab
Regeneron
Angiopoietin-like 3 Inhibitor [EPC]injection2021
Regeneron
PRALUENTApproved
alirocumab
Regeneron
PCSK9 Inhibitor [EPC]injection2015
Amgen
REPATHAApproved
evolocumab
Amgen
PCSK9 Inhibitor [EPC]injection2015

Competitive Landscape

7 companies ranked by most advanced pipeline stage

Regeneron
RegeneronTARRYTOWN, NY
5 programs
1
4
1
AlirocumabPhase 3Monoclonal Antibody
EVKEEZA(Evinacumab)Phase 3Monoclonal Antibody2 trials
evinacumabPhase 3Monoclonal Antibody1 trial
evinacumabPhase 3Monoclonal Antibody1 trial
REGN1500 250 mg SC/15 mg/kg IV/450 mg SCPhase 21 trial
Active Trials
NCT02265952Completed9Est. Jul 2018
NCT06500598Approved For Marketing
NCT04233918Completed20Est. May 2023
+2 more trials
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
1
REPATHA(Evolocumab)Phase 2/3Monoclonal Antibody5 trials
Active Trials
NCT07422285Recruiting400Est. Jul 2026
NCT06140095Unknown8Est. Dec 2025
NCT04951856Active Not Recruiting2,166Est. May 2028
+2 more trials
Arrowhead Pharmaceuticals
3 programs
1
2
ZodasiranPhase 31 trial
zodasiran InjectionPhase 31 trial
ARO-ANG 3 InjectionPhase 21 trial
Active Trials
NCT05217667Active Not Recruiting18Est. Nov 2025
NCT07473843Not Yet Recruiting12Est. May 2029
NCT07037771Recruiting60Est. Aug 2027
Ultragenyx Pharmaceutical
1 program
1
EvinacumabPhase 3Monoclonal Antibody1 trial
Active Trials
NCT05611528Completed10Est. Mar 2025
Ionis Pharmaceuticals
1 program
1
AKCEA-ANGPTL3-LRXPhase 21 trial
Active Trials
NCT03455777Withdrawn0Est. Dec 2018
GS
Gilead SciencesFOSTER CITY, CA
1 program
1
Run-In Period: PlaceboPhase 21 trial
Active Trials
NCT02472535Completed13Est. Feb 2016
AP
Amryt PharmaUK - London
1 program
LomitapideN/A1 trial
Active Trials
NCT02135705Recruiting300Est. Sep 2028

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
AmgenEvolocumab
AmgenEvolocumab
Arrowhead PharmaceuticalsZodasiran
Arrowhead Pharmaceuticalszodasiran Injection
Ultragenyx PharmaceuticalEvinacumab
RegeneronEvinacumab
AmgenEvolocumab
Regeneronevinacumab
Regeneronevinacumab
AmgenEvolocumab
AmgenEvolocumab
AmgenEvolocumab
AmgenEvolocumab
AmgenEvolocumab
AmgenEvolocumab

Showing 15 of 28 trials with date data

Clinical Trials (29)

Total enrollment: 17,280 patients across 29 trials

Evolocumab or Normal Strategies to Reach LDL Objectives in Acute Myocardial Infarction Upbound to PCI

Start: Sep 2021Est. completion: May 20282,166 patients
Phase 4Active Not Recruiting

A Study of Evolocumab on Carotid Artery Atherosclerotic Plaque Morphology Prior to Carotid EndArterectomy

Start: Aug 2020Est. completion: May 202260 patients
Phase 4Unknown

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

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

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

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

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

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

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

Effect of Evolocumab on Coronary Atherosclerosis

Start: Mar 2019Est. completion: May 202355 patients
Phase 3Completed

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

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

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

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

Fourier Open-label Extension Study in Subjects With Clinically Evident Cardiovascular Disease in Selected European Countries

Start: Mar 2017Est. completion: Mar 20221,600 patients
Phase 3Terminated

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)

Start: Sep 2016Est. completion: Jun 2021163 patients
Phase 3Completed

Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment

Start: Dec 2015Est. completion: Jan 201739 patients
Phase 3Completed

Open-label Extension (OLE) Study to Assess Safety and Efficacy of Evolocumab

Start: Nov 2014Est. completion: Mar 2018770 patients
Phase 3Completed

Open-label Extension Study of Evolocumab (AMG 145) in Adults With Hyperlipidemia and Mixed Dyslipidemia

Start: Apr 2013Est. completion: May 20183,681 patients
Phase 3Completed

Study to Assess In-home Use of Evolocumab (AMG 145) Using a Prefilled Syringe or a Prefilled Autoinjector/Pen

Start: Apr 2013149 patients
Phase 3Completed

Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities

Start: Apr 2012Est. completion: Jan 201458 patients
Phase 2/3Completed

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

Start: Apr 2022Est. completion: Nov 202518 patients
Phase 2Active Not Recruiting

Impact of Evolocumab in Cardiac Transplant Patients With CAV

Start: Jul 2019Est. completion: Mar 202326 patients
Phase 2Completed

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

Open Label Study of Long Term Evaluation Against LDL-C Trial

Start: Oct 2011Est. completion: Jun 20181,324 patients
Phase 2Completed

Comparing the Extent to Which Two Evolocumab Drug Products Are Made Available in the Body After a Single Subcutaneous Dose

Start: Jan 2026Est. completion: Jul 2026400 patients
Phase 1Recruiting

Studies to Gain Insight How the Drugs PCSK9-inhibitors and Statins Affect Cholesterol and Bile Acid Metabolism

Start: Feb 2024Est. completion: Dec 20258 patients
Phase 1Unknown

Estimation Study to Assess the Effect of Severe Renal Impairment and End-stage Renal Disease Hemodialysis on the Pharmacokinetics of Evolocumab

Start: Aug 2014Est. completion: Dec 201418 patients
Phase 1Completed

Compassionate Use of Evinacumab

N/AApproved For Marketing

Evolocumab Pregnancy Exposure Registry

Start: Dec 2016Est. completion: Sep 2020140 patients
N/ATerminated

Long Term Post Marketing Specified Drug Use Result Survey for Evolocumab in Japan

Start: Jun 2016Est. completion: Mar 20236,000 patients
N/ACompleted

LOWER: Lomitapide Observational Worldwide Evaluation Registry

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

Phase Legend

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

Key Insights

7 late-stage (Phase 3) programs — potential near-term approvals
3 actively recruiting trials targeting 17,280 patients
7 companies competing in this space