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Familial Chylomicronemia Syndrome

1
Pipeline Programs
2
Companies
9
Clinical Trials
1 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
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 31 trial
Active Trials
NCT05902598Completed37Est. Jan 2026
Ionis Pharmaceuticals
7 programs
OlezarsenN/ARNA Therapeutic2 trials
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
NCT07727538Recruiting12Est. Aug 2031
NCT03360747Completed3Est. Sep 2018
+5 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Ionis PharmaceuticalsOlezarsen
Visirna TherapeuticsPlozasiran
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsOlezarsen
Ionis PharmaceuticalsVolanesorsen
Ionis PharmaceuticalsVolanesorsen
Ionis PharmaceuticalsAKCEA-ANGPTL3-LRx

Clinical Trials (9)

Total enrollment: 337 patients across 9 trials

A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

Start: Jul 2026Est. completion: Aug 203112 patients
Phase 3Recruiting

A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome

Start: Jul 2023Est. completion: Jan 202637 patients
Phase 3Completed

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

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

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

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

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

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

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

N/AApproved For Marketing

Phase Legend

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

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 337 patients
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.