Amyloidosis, Hereditary
2
Pipeline Programs
2
Companies
2
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
0
0
1
1
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
2100%
On Market (1)
Approved therapies currently available
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Ionis PharmaceuticalsCARLSBAD, CA
1 programInotersenN/ARNA Therapeutic1 trial
Active Trials
NCT03400098Approved For Marketing
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Alnylam PharmaceuticalsPatisiran
Clinical Trials (2)
Total enrollment: 164 patients across 2 trials
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Start: Feb 2019Est. completion: Nov 2025164 patients
Phase 3Completed
ATTR Expanded Access Program (EAP) by Ionis
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
2 companies competing in this space