Patisiran

Patisiran
Names
Trade namesOnpattro
Other namesALN-18328
Clinical data
Drug classSmall interfering RNA (siRNA)[1]
Main usesPolyneuropathy in hereditary transthyretin-mediated amyloidosis[1]
Side effectsUpper respiratory infection, shortness of breath, muscle spasms, joint pain, redness, dizziness[1]
Pregnancy
category
  • US: N (Not classified yet)
    Routes of
    use    		Intravenous
    Typical dose300 mcg/kg q 21 days[2]
    External links
    AHFS/Drugs.comMonograph
    US NLMPatisiran
    Legal
    License data
    Legal status
    Chemical and physical data
    FormulaC412H520N148O290P40
    Molar mass13424.388 g·mol−1

    Patisiran, sold under the brand name Onpattro, is a medication used to treat polyneuropathy in people with hereditary transthyretin-mediated amyloidosis.[1] It is given by gradual injection into a vein.[2]

    Common side effects include upper respiratory infection, shortness of breath, muscle spasms, joint pain, redness, and dizziness.[1] Other side effects may include vitamin A deficiency.[1] Safety in pregnancy is unclear; though evidence shows harm in other animals.[1] It is a small piece of RNA that blocks the production of abnormal transthyretin.[3]

    Patisiran was approved for medical use in Europe and the United States in 2018.[3][1] In the United Kingdom a single vial of 10 mg costs the NHS about £7,700 as of 2021.[2] This amount in the United States costs about 10,000 USD.[4]

    Medical uses

    Dosage

    It is given at a dose of 300 mcg/kg every 21 days.[2] The maximum dose is 30 mg.[2]

    Mechanism of action

    It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[5] It utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[6]

    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.[7][8] It was approved for medical use in the United States and in the European Union in August 2018.[9][10]

    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.[11] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[12]

    Society and culture

    Economics

    Patisiran was developed and is marketed by Alnylam.[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]

    As of 2020, there were 1050 patients globally receiving the medication generating $65.5M in net-revenues for Alnylam Pharmaceuticals.[17][18]

    References

    1. 1 2 3 4 5 6 7 8 "Patisiran Monograph for Professionals". Drugs.com. Archived from the original on 26 January 2021. Retrieved 26 October 2021.
    2. 1 2 3 4 5 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1109. ISBN 978-0857114105.
    3. 1 2 "Onpattro". Archived from the original on 8 November 2020. Retrieved 26 October 2021.
    4. ↑ "Onpattro Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 24 June 2021. Retrieved 26 October 2021.
    5. ↑ 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.
    6. ↑ "Onpattro (patisiran)". www.centerwatch.com. Archived from the original on 2021-06-24. Retrieved 2021-06-18.
    7. ↑ "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.
    8. ↑ 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.
    9. ↑ "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.
    10. ↑ "Onpattro EPAR". European Medicines Agency (EMA). Archived from the original on 8 November 2020. Retrieved 2 September 2020.
    11. ↑ 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.
    12. ↑ 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.
    13. ↑ Reidy, Chris (October 22, 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Archived from the original on 7 May 2021. Retrieved 5 May 2021.
    14. ↑ 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. Archived from the original on 2021-10-20. Retrieved 2021-06-24.
    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 2021-06-24. Retrieved 2021-06-23.
    17. ↑ "Patisiran and Vutrisiran, in development for the Treatment of Transthyretin-Mediated Amyloidosis" (PDF). Alnylam Pharmaceuticals. Archived (PDF) from the original on 2021-10-09.
    18. ↑ "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Period Activity". www.businesswire.com. 2021-02-11. Archived from the original on 2021-06-24. Retrieved 2021-06-23.
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