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

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.

Rich TVX News Network Site

Rich TVX News Network Info

© MMXXIII Rich X Search. We shall prevail. All rights reserved. Rich X Search