Patisiran

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]

Patisiran
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

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