Familial Chylomicronemia Syndrome

8
Pipeline Programs
2
Companies
8
Clinical Trials
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
1
0
6
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
6100%
+ 2 programs with unclassified modality

On Market (1)

Approved therapies currently available

Arrowhead Pharmaceuticals
REDEMPLOApproved
plozasiran
Arrowhead Pharmaceuticals
APOC-III-directed RNA Interaction [EPC]subcutaneous2025

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Arrowhead Pharmaceuticals
1 program
1
1
PlozasiranPhase 31 trial
Active Trials
NCT05902598Completed37Est. Jan 2026
Ionis Pharmaceuticals
7 programs
1
5
OlezarsenPhase 3RNA Therapeutic1 trial
OlezarsenPhase 3RNA Therapeutic1 trial
OlezarsenPhase 3RNA Therapeutic1 trial
VolanesorsenPhase 3RNA Therapeutic1 trial
VolanesorsenPhase 3RNA Therapeutic1 trial
+2 more programs
Active Trials
NCT06360237Approved For Marketing
NCT03360747Completed3Est. Sep 2018
NCT05130450Active Not Recruiting60Est. Feb 2028
+4 more trials

Trial Timeline

Clinical trial activity over time

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

Clinical Trials (8)

Total enrollment: 325 patients across 8 trials

NCT05902598Arrowhead PharmaceuticalsPlozasiran

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
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

6 late-stage (Phase 3) programs — potential near-term approvals
RNA Therapeutic is the dominant modality (100% of programs)
2 companies competing in this space