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Patisiran

Alternative Names: patisiran, aln-ttr02, alnttr02, aln ttr02, onpattro, aln-18328, aln18328, aln 18328
Latest Update: 2024-12-14
Latest Update Note: Clinical Trial Update

Product Description

Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/29972753/)

Mechanisms of Action: TTR Modulator

Novel Mechanism: No

Modality: Nucleic Acid

Route of Administration: Intravenous

FDA Designation: None *

Approval Status: Approved

Approved Countries: Australia | Austria | Belgium | Brazil | Canada | Croatia | Cyprus | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Latvia | Lithuania | Luxembourg | Netherlands | Poland | Portugal | Romania | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | United Kingdom | United States

Approved Indications: Transthyretin Amyloidosis | Amyloidosis

Known Adverse Events: Respiratory Tract Infections

Company: Alnylam
Company Location: CAMBRIDGE MA 02142
Company CEO: Yvonne L. Greenstreet
Additonal Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Patisiran

Countries in Clinic: Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Cyprus, Czech Republic, Denmark, France, Germany, Hong Kong, Italy, Japan, Korea, Malaysia, Mexico, Netherlands, New Zealand, Portugal, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States, Unknown Location

Active Clinical Trial Count: 7

Highest Development Phases

Phase 3: Amyloid Neuropathies, Familial|Amyloidosis|Amyloidosis, Familial|Cardiomyopathies|Transthyretin Amyloidosis

Phase 1: Amyloid Neuropathies|Polyneuropathies

Trial

Phase

Trial Status

Disease

Primary Completion Date

Probability of Success

jRCT2031210085

P3

Active, not recruiting

Amyloidosis

2025-07-31

ALN-TTR02-011

P3

Unknown Status

Transthyretin Amyloidosis

2025-06-30

001 amendment 02

P1

Active, not recruiting

Amyloid Neuropathies|Polyneuropathies|Transthyretin Amyloidosis

2025-06-30

A study to Evaluate Patisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy

P3

Active, not recruiting

Transthyretin Amyloidosis

2023-10-28

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