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
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 |