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Synonyms: ALN-18328 | ALN-TTR02 | Onpattro®
patisiran is an approved drug (EMA & EMA & FDA (2018), UK MHRA (2021))
Compound class:
Nucleic acid
Comment: Patisiran (ALN-TTR02) is a double-stranded small interfering RNA (siRNA) class drug [2]. It reduces hepatic production of mutated transthyretin protein, as a mechanism to treat hereditary transthyretin amyloidosis with polyneuropathy [1,3-4]. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
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| No information available. |
Summary of Clinical Use  |
| Patisiran was approved by both the EMA and FDA in August 2018, to treat polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) [5]. Authorisation for use in the UK followed in 2021. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT03759379 | HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| NCT03862807 | Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| NCT03997383 | APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| External links |
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For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA) |
