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Transthyretin Amyloidosis

4
Pipeline Programs
8
Companies
8
Clinical Trials
4 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
3
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
150%
Peptide
150%
+ 11 programs with unclassified modality

Competitive Landscape

8 companies ranked by most advanced pipeline stage

Attralus
AttralusFL - Naples
1 program
1
I 124-EvuzamitidePhase 2Peptide1 trial
Active Trials
NCT05635045Completed10Est. Jun 2024
Alnylam Pharmaceuticals
5 programs
2
ATTR Algorithm Identification StudyPhase 1
Computer algorithm for ATTRPhase 11 trial
99mTc-pyrophosphate ScintigraphyN/A
ATTR Prevalence and Prediction StudyN/A
TRAMmoniTTR TTR Screening and Monitoring StudyN/A
Active Trials
NCT04569903Withdrawn0Est. Feb 2025
Corino Therapeutics
Corino TherapeuticsNC - Charlottesville
1 program
1
TolcaponePhase 11 trial
Active Trials
NCT03591757Completed10Est. Apr 2019
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
2 programs
The Eplontersen Pregnancy and Lactation Outcomes StudyN/ARNA Therapeutic1 trial
Treatment of transthyretinN/A1 trial
Active Trials
NCT07205666Recruiting10Est. Dec 2035
NCT06465810Recruiting1,850Est. Dec 2031
Pfizer
PfizerNEW YORK, NY
1 program
99mTc-pyrophosphate ScintigraphyN/A
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
1 program
99mTc-pyrophosphate ScintigraphyN/A1 trial
Active Trials
NCT05577819Recruiting515Est. Dec 2026
CENTOGENE
CENTOGENEGermany - Rostock
1 program
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)N/A1 trial
Active Trials
NCT02713880Withdrawn0Est. Dec 2019
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
1 program
Phenotyping and Characterization of wtATTR-CM (TRACE 1)N/A1 trial
Active Trials
NCT06291805Recruiting120Est. Dec 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
AttralusI 124-Evuzamitide
Alnylam PharmaceuticalsComputer algorithm for ATTR
Corino TherapeuticsTolcapone
AstraZenecaThe Eplontersen Pregnancy and Lactation Outcomes Study
AstraZenecaTreatment of transthyretin
Novo NordiskPhenotyping and Characterization of wtATTR-CM (TRACE 1)
BridgeBio Pharma99mTc-pyrophosphate Scintigraphy
CENTOGENEBiomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)

Clinical Trials (8)

Total enrollment: 2,515 patients across 8 trials

NCT05635045AttralusI 124-Evuzamitide

Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR

Start: Jul 2022Est. completion: Jun 202410 patients
Phase 2Completed
NCT04569903Alnylam PharmaceuticalsComputer algorithm for ATTR

Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records

Start: Aug 2022Est. completion: Feb 20250
Phase 1Withdrawn

Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)

Start: Oct 2018Est. completion: Apr 201910 patients
Phase 1Completed
NCT07205666AstraZenecaThe Eplontersen Pregnancy and Lactation Outcomes Study

The Eplontersen Pregnancy and Lactation Outcomes Study

Start: Nov 2025Est. completion: Dec 203510 patients
N/ARecruiting
NCT06465810AstraZenecaTreatment of transthyretin

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Start: Jun 2024Est. completion: Dec 20311,850 patients
N/ARecruiting
NCT06291805Novo NordiskPhenotyping and Characterization of wtATTR-CM (TRACE 1)

Phenotyping and Characterization of wtATTR-CM (TRACE 1)

Start: Feb 2024Est. completion: Dec 2025120 patients
N/ARecruiting
NCT05577819BridgeBio Pharma99mTc-pyrophosphate Scintigraphy

Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF

Start: Oct 2020Est. completion: Dec 2026515 patients
N/ARecruiting
NCT02713880CENTOGENEBiomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)

Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)

Start: Aug 2018Est. completion: Dec 20190
N/AWithdrawn

Related Jobs

Phase Legend

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

Key Insights

4 actively recruiting trials targeting 2,515 patients
8 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.