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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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| References |
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1. Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL et al.. (2018)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med, 379 (1): 11-21. [PMID:29972753] |
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2. Bennett CF, Baker BF, Pham N, Swayze E, Geary RS. (2017)
Pharmacology of Antisense Drugs. Annu Rev Pharmacol Toxicol, 57: 81-105. [PMID:27732800] |
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3. Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E et al.. (2013)
Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med, 369 (9): 819-29. [PMID:23984729] |
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4. Suhr OB, Coelho T, Buades J, Pouget J, Conceicao I, Berk J, Schmidt H, Waddington-Cruz M, Campistol JM, Bettencourt BR et al.. (2015)
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis, 10: 109. [PMID:26338094] |
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5. Wood H. (2018)
FDA approves patisiran to treat hereditary transthyretin amyloidosis. Nat Rev Neurol, 14 (10): 570. [PMID:30158559] |
