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Familial Chylomicronemia Syndrome

1
Pipeline Programs
2
Companies
7
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
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
6100%
+ 2 programs with unclassified modality

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Visirna Therapeutics
Visirna TherapeuticsChina - Shanghai
1 program
1
PlozasiranPhase 3
Ionis Pharmaceuticals
7 programs
OlezarsenN/ARNA Therapeutic1 trial
AKCEA-ANGPTL3-LRxPHASE_21 trial
OlezarsenPHASE_3RNA Therapeutic1 trial
OlezarsenPHASE_3RNA Therapeutic1 trial
OlezarsenPHASE_3RNA Therapeutic1 trial
+2 more programs
Active Trials
NCT06360237Approved For Marketing
NCT03360747Completed3Est. Sep 2018
NCT04568434Completed66Est. Oct 2023
+4 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsVolanesorsen
Ionis PharmaceuticalsVolanesorsen
Ionis PharmaceuticalsAKCEA-ANGPTL3-LRx

Clinical Trials (7)

Total enrollment: 288 patients across 7 trials

NCT05185843Ionis PharmaceuticalsOlezarsen

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

Start: Feb 2022Est. completion: Jun 202724 patients
Phase 3Active Not Recruiting
NCT05130450Ionis PharmaceuticalsOlezarsen

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Start: Nov 2021Est. completion: Feb 202860 patients
Phase 3Active Not Recruiting
NCT04568434Ionis PharmaceuticalsOlezarsen

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)

Start: Nov 2020Est. completion: Oct 202366 patients
Phase 3Completed
NCT02658175Ionis PharmaceuticalsVolanesorsen

The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome

Start: Dec 2015Est. completion: Jan 202068 patients
Phase 3Completed
NCT02211209Ionis PharmaceuticalsVolanesorsen

The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

Start: Dec 2014Est. completion: Mar 201767 patients
Phase 3Completed
NCT03360747Ionis PharmaceuticalsAKCEA-ANGPTL3-LRx

Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

Start: Dec 2017Est. completion: Sep 20183 patients
Phase 2Completed
NCT06360237Ionis PharmaceuticalsOlezarsen

Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

N/AApproved For Marketing

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
RNA Therapeutic is the dominant modality (100% of programs)
2 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.