Back باتيسيران Arabic Patisiran German Patisiran Spanish Patisiran French פטיסיראן HE パチシラン Japanese ପାଟିସିରାନ OR Patisirana Portuguese Патисиран Russian Патісіран Ukrainian

Patisiran

Patisiran
Clinical data
Trade namesOnpattro
Other namesALN-18328
AHFS/Drugs.comMonograph
License data
Pregnancy
category
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC412H520N148O290P40
Molar mass13424.388 g·mol−1

Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis,[7] a fatal rare disease that is estimated to affect 50,000 people worldwide.[8][9]

It is the first small interfering RNA-based drug approved by the U.S. Food and Drug Administration (FDA) and the first drug approved by the FDA to treat this condition.[8] It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[10] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[11]

Patisiran was developed and is marketed by Alnylam.[12] The FDA considers it to be a first-in-class medication.[13]

  1. ^ a b "Onpattro". Therapeutic Goods Administration (TGA). 2 December 2022. Archived from the original on 5 February 2023. Retrieved 28 April 2023.
  2. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 21 December 2022. Archived from the original on 3 April 2022. Retrieved 2 January 2023.
  3. ^ "Onpattro patisiran 10 mg/5 mL concentrated injection for infusion vial (380813)". Therapeutic Goods Administration (TGA). 22 November 2022. Archived from the original on 18 December 2022. Retrieved 28 April 2023.
  4. ^ "Onpattro (Alnylam Australia Pty Ltd)". Therapeutic Goods Administration (TGA). 5 December 2022. Archived from the original on 27 March 2023. Retrieved 28 April 2023.
  5. ^ "Onpattro PI" (PDF). Archived from the original (PDF) on 30 May 2022.
  6. ^ "Summary Basis of Decision (SBD) for Onpattro". Health Canada. 23 October 2014. Archived from the original on 31 May 2022. Retrieved 29 May 2022.
  7. ^ a b "Onpattro- patisiran injection, lipid complex". DailyMed. U.S. National Library of Medicine. 10 May 2021. Archived from the original on 4 May 2022. Retrieved 9 June 2022.
  8. ^ a b Loftus P (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. Archived from the original on 10 August 2018. Retrieved 10 August 2018.
  9. ^ Elafros MA, Kvalsund MP, Callaghan BC (June 2022). "The Global Burden of Polyneuropathy-In Need of an Accurate Assessment". JAMA Neurology. 79 (6): 537–538. doi:10.1001/jamaneurol.2022.0565. PMC 9197927. PMID 35404377. S2CID 248083689.
  10. ^ Kristen AV, Ajroud-Driss S, Conceição I, Gorevic P, Kyriakides T, Obici L (February 2019). "Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis". Neurodegenerative Disease Management. 9 (1): 5–23. doi:10.2217/nmt-2018-0033. PMID 30480471.
  11. ^ "Onpattro (patisiran)". www.centerwatch.com. Archived from the original on 24 June 2021. Retrieved 18 June 2021.
  12. ^ Reidy C (22 October 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Archived from the original on 7 May 2021. Retrieved 5 May 2021.
  13. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.

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