Patisiran

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Patisiran
Patisiran.png
Clinical data
Trade namesOnpattro
Other namesALN-18328
AHFS/Drugs.comMonograph
License data
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 for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide.[1]

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.[1] It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[2] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[3]

Patisiran was developed and is marketed by Alnylam.[4] The FDA considers it to be a first-in-class medication.[5]

Global use[]

As of 2020, there were 1050 patients globally receiving Onpattro, generating $65.5M in net-revenues for Alnylam Pharmaceuticals.[6][7]

History[]

Patisiran was granted orphan drug status, fast track designation, priority review and breakthrough therapy designation due to its novel mechanism and the rarity of the condition it treats.[8][9] It was approved for medical use in the United States and in the European Union in August 2018.[10][11] The per-patient cost is between US$451,430 and US$677,145 per year, depending on the number of vials needed.[12][13][14]

References[]

  1. ^ a b Loftus P (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. Retrieved 10 August 2018.
  2. ^ Kristen, Arnt V; Ajroud-Driss, Senda; Conceição, Isabel; Gorevic, Peter; Kyriakides, Theodoros; Obici, Laura (2018-11-27). "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. ISSN 1758-2024. PMID 30480471.
  3. ^ "Onpattro (patisiran)". www.centerwatch.com. Retrieved 2021-06-18.
  4. ^ Reidy, Chris (October 22, 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Retrieved 5 May 2021.
  5. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
  6. ^ "Patisiran and Vutrisiran, in development for the Treatment of Transthyretin-Mediated Amyloidosis" (PDF). Alnylam Pharmaceuticals.{{cite web}}: CS1 maint: url-status (link)
  7. ^ "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Period Activity". www.businesswire.com. 2021-02-11. Retrieved 2021-06-23.
  8. ^ "FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease" (Press release). U.S. Food and Drug Administration (FDA). 10 August 2018. Retrieved 11 August 2018.
  9. ^ Brooks M (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Retrieved 10 August 2018.
  10. ^ "Drug Approval Package: Onpattro (patisiran)". U.S. Food and Drug Administration (FDA). 7 September 2018. Retrieved 2 September 2020.
  11. ^ "Onpattro EPAR". European Medicines Agency (EMA). Retrieved 2 September 2020.
  12. ^ Information, National Center for Biotechnology; Pike, U. S. National Library of Medicine 8600 Rockville; MD, Bethesda (2019-08-01). Executive Summary. Canadian Agency for Drugs and Technologies in Health.
  13. ^ Lipschultz B, Cortez M (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Retrieved 11 August 2018.
  14. ^ "Onpattro Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 2021-06-23.

External links[]

  • "Patisiran". Drug Information Portal. U.S. National Library of Medicine.
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