Drug

Patisiran

In clinical development

ClassTransthyretin mRNA RNAi inhibitorEmerging researchLate-stage development

Also known as Onpattro.

RxNorm2053490 ChEMBLCHEMBL3989987 ATCN07XX12UNII50FKX8CB2Y

Research papers

Protein targets

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Clinical trial2026-04-20Results posted · ClinicalTrials.gov

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

Clinical trial2026-04-13Completed · ClinicalTrials.gov

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Clinical trial2026-01-12Results posted · ClinicalTrials.gov

APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

Clinical trial2024-04-22Results posted · ClinicalTrials.gov

An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant

Clinical trial2024-04-22Results posted · ClinicalTrials.gov

A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis

Clinical trial2024-04-19Results posted · ClinicalTrials.gov

A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study

Clinical trial2023-12-06Results posted · ClinicalTrials.gov

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

Research2023-10-01The New England journal of medicine

Therapeutic siRNA: state of the art.

Research2020-06-19Signal transduction and targeted therapy

A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers

Clinical trial2014-06-01Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Patisiran

Aliases & brands

Onpattro

RxNorm CUI

2053490

ChEMBL ID

CHEMBL3989987

ATC codes

N07XX12

UNII

50FKX8CB2Y

Primary mechanism

Transthyretin mRNA RNAi inhibitor

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

ENSG00000118271

RNAI INHIBITORTransthyretin mRNA RNAi inhibitor

Clinical trials

8 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme

CLINICALTRIALS.GOV · LIVE REGISTRY

registered trials across all phases

LATEST COMPLETION 2026

Late-stage (III+)

Completed

PHASE DISTRIBUTIONn = 8

Early Phase 11Phase 11Phase 21Phase 35

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

CompletedPH IIIAlnylam Pharmaceuticalsn=3602025

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

CompletedPH IIIAlnylam Pharmaceuticalsn=1642025

CompletedPH IIIAlnylam Pharmaceuticalsn=2112022

CompletedPH IIIAlnylam Pharmaceuticalsn=242020

CompletedPH IIIAlnylam Pharmaceuticalsn=2252017

Recent completions

Trials that read out recently, adding to the completed evidence base.

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

CompletedPH IAustin Neuromuscular Centern=102026

CompletedPH IIIAlnylam Pharmaceuticalsn=3602025

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

CompletedPH IIIAlnylam Pharmaceuticalsn=1642025

CompletedPH IIIAlnylam Pharmaceuticalsn=2112022

CompletedPH IIIAlnylam Pharmaceuticalsn=242020

CompletedPH IIIAlnylam Pharmaceuticalsn=2252017

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time

20202023

Most influential

Therapeutic siRNA: state of the art.

Signal transduction and targeted therapy · 2020 · 991 cites

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.

The New England journal of medicine · 2023 · 230 cites

Related drugs

2 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Acetylgalactosamine

Studied across the same disease area as Patisiran in the shared literature.

1 shared disease1 shared paper

Givosiran

Studied across the same disease area as Patisiran in the shared literature.

1 shared disease1 shared paper

References & data sources

RxNorm (U.S. National Library of Medicine) — drug identity
ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
Europe PMC — research literature
ClinicalTrials.gov — clinical trials