Patisiran

Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis,[3] a fatal rare disease that is estimated to affect 50,000 people worldwide.[4][5]

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.[4] It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[6] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[7]

Patisiran was developed and is marketed by Alnylam.[8] The FDA considers it to be a first-in-class medication.[9]

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.[10][11] It was approved for medical use in the United States and in the European Union in August 2018.[12][13] The per-patient cost is between US$451,430 and US$677,145 per year, depending on the number of vials needed.[14][15][16]

Formulation

The siRNA active component of Patisiran is formulated into lipid nanoparticles, which protect the RNA and facilitate its delivery to target tissues. The lipid nanoparticle formulation includes buffer components, as well as the lipid components DLin-MC3-DMA, Distearoylphosphatidylcholine, cholesterol, and the PEGylated lipid DMG-PEG 2000.[17][18]

Society and culture

Economics

As of 2020, there were 1050 people globally receiving patisiran, generating $65.5M in net-revenues for Alnylam Pharmaceuticals.[19][20]

References
  1. "PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION ONPATTRO®" (PDF). Archived from the original (PDF) on 30 May 2022.
  2. "Summary Basis of Decision (SBD) for Onpattro". Health Canada. 23 October 2014. Archived from the original on 31 May 2022. Retrieved 29 May 2022.
  3. "Onpattro- patisiran injection, lipid complex". DailyMed. 10 May 2021. Archived from the original on 4 May 2022. Retrieved 9 June 2022.
  4. 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.
  5. Elafros, Melissa A.; Kvalsund, Michelle P.; Callaghan, Brian C. (11 April 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.
  6. Kristen, Arnt V; Ajroud-Driss, Senda; Conceição, Isabel; Gorevic, Peter; Kyriakides, Theodoros; Obici, Laura (27 November 2018). "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.
  7. "Onpattro (patisiran)". www.centerwatch.com. Archived from the original on 24 June 2021. Retrieved 18 June 2021.
  8. Reidy, Chris (22 October 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Archived from the original on 7 May 2021. Retrieved 5 May 2021.
  9. 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.
  10. "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. Archived from the original on 7 September 2018. Retrieved 11 August 2018.
  11. Brooks M (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Archived from the original on 4 July 2019. Retrieved 10 August 2018.
  12. "Drug Approval Package: Onpattro (patisiran)". U.S. Food and Drug Administration (FDA). 7 September 2018. Archived from the original on 12 April 2021. Retrieved 2 September 2020.
  13. "Onpattro EPAR". European Medicines Agency (EMA). Archived from the original on 8 November 2020. Retrieved 2 September 2020.
  14. Information, National Center for Biotechnology; Pike, U. S. National Library of Medicine 8600 Rockville; MD, Bethesda (1 August 2019). Executive Summary. Canadian Agency for Drugs and Technologies in Health. Archived from the original on 20 October 2021. Retrieved 3 April 2021.
  15. Lipschultz B, Cortez M (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Archived from the original on 14 November 2019. Retrieved 11 August 2018.
  16. "Onpattro Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 24 June 2021. Retrieved 23 June 2021.
  17. 1. Akinc, A. et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat. Nanotechnol. 14, 1084–1087 (2019).
  18. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210922s000lbl.pdf
  19. "Patisiran and Vutrisiran, in development for the Treatment of Transthyretin-Mediated Amyloidosis" (PDF). Alnylam Pharmaceuticals. Archived (PDF) from the original on 9 October 2021.
  20. "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Period Activity" (Press release). Alnylam. 11 February 2021. Archived from the original on 24 June 2021. Retrieved 23 June 2021 – via Business Wire.
  • "Patisiran". Drug Information Portal. U.S. National Library of Medicine.
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