Friday, July 10, 2026

Drug profile · INN

patisiran

patisiran (ONPATTRO) is a transthyretin mrna rnai inhibitor. Associated with Lacuna Pharma Pty Ltd.

EMA: approved TGA: approved Nervous system (N07) N07
Patents linked 0
Pipeline programs 10
News articles 0
Drug details — patisiran
Class
Nervous system (N07)
Route
Patents
0
Programs
10
Data quality
0.89

Quick answer

patisiran (ONPATTRO) is a transthyretin mrna rnai inhibitor. Associated with Lacuna Pharma Pty Ltd.

Key facts

INN patisiran
Brand ONPATTRO
Mechanism Transthyretin mRNA RNAi inhibitor
Target Transthyretin mRNA
Therapeutic class Nervous system (N07)
ATC code N07

Mechanism of action

Transthyretin mRNA RNAi inhibitor

Primary target: Transthyretin mRNA

Approval history

Authority Status Date
EMA approved
TGA approved

Pipeline programs

  1. ALN-TTR02-011 Phase 3 · Alnylam Netherlands · Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloido
  2. ALN-TTRSC02-002 Phase 3 · Alnylam Netherlands · Hereditary Transthyretin Amyloidosis
  3. Patisiran Phase 3 · Alnylam Netherlands · Amyloidosis
  4. Patisiran Phase 3 · Alnylam Netherlands · Amyloidosis, Familial
  5. Patisiran Phase 3 · Alnylam Netherlands · Amyloidosis, Hereditary
  6. patisiran (ALN-TTR02) Phase 3 · Alnylam Netherlands · TTR-mediated Amyloidosis
  7. ALN-TTR02 (patisiran) administered by intravenous (IV) infusion Phase 2 · Alnylam Netherlands · TTR-mediated Amyloidosis
  8. Patisiran Phase 2 · Alnylam Netherlands · TTR-mediated Amyloidosis

Related & competitor drugs

  1. aripiprazole Nervous system (N05)
  2. brexpiprazole Nervous system (N05)
  3. brivaracetam Nervous system (N03)
  4. bupivacaine Nervous system (N01)
  5. buprenorphine Nervous system (N07)
  6. caffeine citrate Nervous system (N06)

Frequently asked questions

What is patisiran?

patisiran (ONPATTRO) is a transthyretin mrna rnai inhibitor. Associated with Lacuna Pharma Pty Ltd.

What is the brand name for patisiran?

ONPATTRO is a marketed brand name for patisiran. Check FDA Drugs@FDA for the current US label holder and prescribing information.