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Patisiran parenteral

Updated 2 Feb 2023 | Transthyretin amyloidosis


Infusions of patisiran.

Drugs List

  • ONPATTRO 10mg/5ml concentrate for solution for infusion vial
  • patisiran 10mg/5ml concentrate for solution for infusion vial
  • Therapeutic Indications


    Polyneuropathy in patients with hereditary transthyretin amyloidosis

    Treatment of hereditary transthyretin mediated amyloidosis in adult patients with stage one or two polyneuropathy.



    300micrograms/kg once every three weeks.

    Maximum dose of 30mg for patients weighing more than or equal to 100kg.

    Additional Dosage Information

    To reduce the risk of infusion related reactions (IRR), the following medicinal products should be given at least 60 minutes before infusion of patisiran:

    Intravenous corticosteroid (dexamethasone 10mg, or equivalent)
    Oral paracetamol 500mg
    Intravenous H1 blocker (diphenhydramine 50mg, or equivalent)
    Intravenous H2 blocker (ranitidine 50mg, or equivalent)

    The corticosteroid may be tapered in decrements no greater than 2.5mg to a minimum of 5mg dexamethasone. Before each reduction in corticosteroid premedication, the patient should receive 3 consecutive IV infusions of patisiran without experiencing IRRs.

    If necessary additional or higher doses of one or more of the premedications may be administered to reduce the risk of IRRs.

    Vitamin A supplementation
    2500 units once a day.

    Missed doses
    A missed dose of patisiran should be administered as soon as possible.

    If administered within 3 days of a missed dose, the dosing schedule should be continued according to the original schedule.
    If administered more than 3 days after the missed dose, dosing should be continued every 3 weeks thereafter.


    For intravenous use only.

    Patisiran should be diluted prior to use.


    Children under 18 years

    Precautions and Warnings

    Restricted sodium intake
    End stage renal disease
    Liver transplant
    Moderate hepatic impairment
    Severe renal impairment

    Sodium content of formulation may be significant
    Consider premedication with antihistamine and/or corticosteroid
    Correct vitamin A deficiency before starting treatment
    Treatment to be initiated and supervised by a specialist
    Vitamin A supplement is advised during treatment
    Must be diluted before use
    Exclude pregnancy prior to initiation of treatment
    Discontinue treatment immediately if pregnancy is suspected
    Interrupt therapy/reduce infusion rate if infusion-related reactions occur
    May affect results of some laboratory tests
    Discontinue permanently if life threatening infusion reactions occur
    Female: Ensure adequate contraception during treatment

    Treatment may lead to a decrease in vitamin A levels, ocular symptoms of vitamin A deficiency should be evaluated and corrected before treatment is initiated.

    Abnormal vitamin A levels is associated with an increased risk of foetal malformation. Treatment should be discontinued if the women intends to become pregnant and vitamin A levels should be returned to normal before conception is attempted. Supplementation of vitamin A should be discontinued unless the women is vitamin A deficient.

    Pregnancy and Lactation


    Patisiran is contraindicated during pregnancy.

    The manufacturer advises not using patisiran during pregnancy unless the clinical condition of the women requires treatment. Animal studies are insufficient regarding reproductive toxicity. At the time of writing there is limited published information regarding the use of patisiran during pregnancy. The potential risks are unknown. In the event of an unplanned pregnancy, vitamin A and thyroid stimulating hormone (TSH) levels should be taken early in the pregnancy, and the foetus should be monitored carefully particularly during the first trimester.


    Patisiran is contraindicated during breastfeeding.

    The manufacturer advises that the patient either discontinues patisiran or discontinues breastfeeding. It is unknown if patisiran is excreted in human milk. Animal studies have shown small amounts of patisiran are excreted in milk. The effects on exposed infants are unknown.

    Side Effects

    Abdominal pain
    Burning sensation
    Chest discomfort
    Chest pain
    Facial oedema
    Increased heart rate
    Infusion related reaction
    Local pain (injection site)
    Muscle spasm
    Musculoskeletal pain
    Peripheral oedema
    Swelling (injection site)

    Effects on Laboratory Tests

    Laboratory tests for serum vitamin A should not be used to guide vitamin A supplementation during treatment. Readings may not reflect the total amount of vitamin A in the body.


    It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

    The following number will direct the caller to the relevant local centre (0844) 892 0111

    Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( ) or if this is unavailable at the backup site ( ).

    Further Information

    Last Full Review Date: September 2020

    Reference Sources

    Summary of Product Characteristics: Onpattro 2mg/ml concentrate for solution for infusion. Alnylam UK Ltd. Revised November 2020.

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    Medscape UK | Univadis prescription drug monographs & interactions are based on FDB Multilex Content

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    FDB Disclaimer : FDB Multilex is intended for the use of healthcare professionals and is provided on the basis that the healthcare professionals will retain FULL and SOLE responsibility for deciding what treatment to prescribe or dispense for any particular patient or circumstance.