Familial Partial Lipodystrophy
4
Pipeline Programs
3
Companies
4
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
2
1
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 3 programs with unclassified modality
Competitive Landscape
3 companies ranked by most advanced pipeline stage
AP
Ionis PharmaceuticalsCARLSBAD, CA
2 programs1
1
volanesorsenPhase 2/3RNA Therapeutic1 trial
AKCEA-ANGPTL3-LRxPhase 21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
Amryt PharmaMetreleptin
Ionis Pharmaceuticalsvolanesorsen
RegeneronREGN4461
Ionis PharmaceuticalsAKCEA-ANGPTL3-LRx
Clinical Trials (4)
Total enrollment: 88 patients across 4 trials
Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
Start: Oct 2024Est. completion: Dec 202824 patients
Phase 3Recruiting
The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
Start: Dec 2015Est. completion: Nov 201940 patients
Phase 2/3Terminated
A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD)
Start: Feb 2022Est. completion: Apr 202420 patients
Phase 2Terminated
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Start: Jun 2018Est. completion: Aug 20194 patients
Phase 2Completed
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
1 actively recruiting trials targeting 88 patients
3 companies competing in this space