Biology:Patisiran

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Short description: Pharmaceutical drug
Patisiran
Patisiran sodium.svg
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]

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.[14][15] It was approved for medical use in the United States and in the European Union in August 2018.[16][17] The per-patient cost is between US$451,430 and US$677,145 per year, depending on the number of vials needed.[18]

[19][20]

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.[21]

Society and culture

Economics

As of 2020, there were 1050 people globally receiving patisiran, generating $65.5M in net-revenues for Alnylam Pharmaceuticals.[22][23]

References

  1. ↑ 1.0 1.1 "Onpattro". 2 December 2022. https://www.tga.gov.au/resources/auspmd/onpattro. 
  2. ↑ "Updates to the Prescribing Medicines in Pregnancy database". 21 December 2022. https://www.tga.gov.au/resources/resource/guidance/updates-prescribing-medicines-pregnancy-database. 
  3. ↑ "Onpattro patisiran 10 mg/5 mL concentrated injection for infusion vial (380813)". 22 November 2022. https://www.tga.gov.au/resources/artg/380813. 
  4. ↑ "Onpattro (Alnylam Australia Pty Ltd)". 5 December 2022. https://www.tga.gov.au/resources/prescription-medicines-registrations/onpattro-alnylam-australia-pty-ltd. 
  5. ↑ "Onpattro PI". https://pdf.hres.ca/dpd_pm/00060392.PDF. 
  6. ↑ "Summary Basis of Decision (SBD) for Onpattro". 23 October 2014. https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00446&lang=en. 
  7. ↑ 7.0 7.1 "Onpattro- patisiran injection, lipid complex". DailyMed. U.S. National Library of Medicine. 10 May 2021. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e87ec36f-b4b4-49d4-aea4-d4ffb09b0970. 
  8. ↑ 8.0 8.1 "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. 10 August 2018. https://www.wsj.com/articles/fda-approves-first-drug-based-on-gene-silencing-research-1533923359. 
  9. ↑ "The Global Burden of Polyneuropathy-In Need of an Accurate Assessment". JAMA Neurology 79 (6): 537–538. June 2022. doi:10.1001/jamaneurol.2022.0565. PMID 35404377. 
  10. ↑ "Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis". Neurodegenerative Disease Management 9 (1): 5–23. February 2019. doi:10.2217/nmt-2018-0033. PMID 30480471. 
  11. ↑ "Onpattro (patisiran)". https://www.centerwatch.com/directories/1067-fda-approved-drugs/listing/3946-onpattro-patisiran. 
  12. ↑ "Alnylam, Genzyme Form Alliance". The Boston Globe. 22 October 2012. https://www.bostonglobe.com/business/2012/10/22/alnylam-genzyme-form-alliance/hPpfyV15lwB2QlDJbSssBL/story.html. 
  13. ↑ (PDF) New Drug Therapy Approvals 2018 (Report). January 2019. https://www.fda.gov/media/120357/download. Retrieved 16 September 2020. 
  14. ↑ "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.
  15. ↑ "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". WebMD. 10 August 2018. https://www.medscape.com/viewarticle/900541. 
  16. ↑ "Drug Approval Package: Onpattro (patisiran)". 7 September 2018. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/210922Orig1s000TOC.cfm. 
  17. ↑ "Onpattro EPAR". 17 September 2018. https://www.ema.europa.eu/en/medicines/human/EPAR/onpattro. 
  18. ↑ "Executive Summary". Pharmacoeconomic Review Report: Patisiran (Onpattro) (Alnylam Netherlands B.V.): Indication: Treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health. August 2019. https://www.ncbi.nlm.nih.gov/books/NBK549697/. 
  19. ↑ "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". 10 August 2018. https://www.bloomberg.com/news/articles/2018-08-10/alnylam-wins-first-u-s-drug-approval-in-rare-genetic-disease. 
  20. ↑ "Onpattro Prices, Coupons & Patient Assistance Programs". https://www.drugs.com/price-guide/onpattro. 
  21. ↑ "The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs". Nature Nanotechnology 14 (12): 1084–1087. December 2019. doi:10.1038/s41565-019-0591-y. PMID 31802031. Bibcode: 2019NatNa..14.1084A. https://pure.tue.nl/ws/files/142146193/s41565_019_0591_y.pdf. 
  22. ↑ "Patisiran and Vutrisiran, in development for the Treatment of Transthyretin-Mediated Amyloidosis". https://www.alnylam.com/wp-content/uploads/2020/09/2020-RNAi-Roundtable_TTR_FINAL.pdf. 
  23. ↑ "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Period Activity" (Press release). Alnylam. 2021-02-11. Archived from the original on 24 June 2021. Retrieved 2021-06-23 – via Business Wire.



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