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TTR-mediated Amyloidosis

4
Pipeline Programs
1
Companies
19
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 2 programs with unclassified modality

On Market (1)

Approved therapies currently available

Alnylam Pharmaceuticals
ONPATTROApproved
patisiran
Alnylam Pharmaceuticals
intravenous2018
9M Part D

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
3 programs
1
2
1
ONPATTRO(patisiran)Phase 3RNA Therapeutic5 trials
revusiranPhase 35 trials
ALN-TTR02Phase 15 trials
Active Trials
NCT02939820Approved For Marketing
NCT02510261Completed211Est. Nov 2022
NCT02053454Completed12Est. Jun 2014
+12 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Alnylam Pharmaceuticalspatisiran
Alnylam Pharmaceuticalspatisiran
Alnylam Pharmaceuticalspatisiran
Alnylam PharmaceuticalsALN-TTR02
Alnylam Pharmaceuticalsrevusiran
Alnylam PharmaceuticalsALN-TTR02
Alnylam Pharmaceuticalsrevusiran
Alnylam Pharmaceuticalsrevusiran
Alnylam Pharmaceuticalsrevusiran
Alnylam PharmaceuticalsALN-TTR02
Alnylam PharmaceuticalsALN-TTR02
Alnylam Pharmaceuticalspatisiran
Alnylam PharmaceuticalsALN-TTR02
Alnylam Pharmaceuticalsrevusiran
Alnylam PharmaceuticalsALN-TTR02

Showing 15 of 17 trials with date data

Clinical Trials (19)

Total enrollment: 1,510 patients across 19 trials

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Start: Sep 2019Est. completion: Dec 2025360 patients
Phase 3Completed

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Start: Mar 2019Est. completion: Oct 202024 patients
Phase 3Completed

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

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Start: Jul 2015Est. completion: Nov 2022211 patients
Phase 3Completed

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Start: Dec 2014Est. completion: Mar 2017206 patients
Phase 3Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Start: Nov 2013Est. completion: Aug 2017225 patients
Phase 3Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

Start: Oct 2015Est. completion: Feb 201712 patients
Phase 2Completed

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Start: Oct 2014Est. completion: Feb 201725 patients
Phase 2Completed

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Start: Dec 2013Est. completion: Jan 201526 patients
Phase 2Completed

The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Start: Oct 2013Est. completion: Aug 201627 patients
Phase 2Completed

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

Start: May 2012Est. completion: Jan 201429 patients
Phase 2Completed

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

Start: Aug 2022Est. completion: Feb 202610 patients
Phase 1Active Not Recruiting

A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

Start: Jan 2014Est. completion: Jun 201412 patients
Phase 1Completed

A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers

Start: Mar 2013Est. completion: May 201585 patients
Phase 1Completed

Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

Start: Mar 2012Est. completion: Nov 201217 patients
Phase 1Completed

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

N/ANo Longer Available

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

N/AApproved For Marketing

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Start: Aug 2020Est. completion: Oct 203010 patients
N/ARecruiting

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Start: Dec 2019Est. completion: May 202267 patients
N/ACompleted

Phase Legend

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

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 1,510 patients
1 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.