Drug Action
Patisiran is a double-stranded small interfering RNA which reduces transthyretin production.
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Drug reference
Patisiran is a double-stranded small interfering RNA which reduces transthyretin production.
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Stage 1 or stage 2 polyneuropathy in patients with hereditary transthyretin amyloidosis (hATTR) (initiated under specialist supervision) ▶BY INTRAVENOUS INFUSION ▶Adult: 300 micrograms/kg every 3 weeks (max. per dose 30 mg), dosage is based on actual body weight, for information on premedication, vitamin A supplementation, and missed doses—consult product literature
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Manufacturer advises plasma vitamin A levels below the lower limit of normal should be corrected and any ocular symptoms or signs of vitamin A deficiency should be evaluated before starting treatment.
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Manufacturer advises patients should receive pre- medication (to reduce the risk of infusion-related reactions)—consult product literature. l HANDLING AND STORAGE Manufacturer advises store in a refrigerator (2–8°C)—consult product literature for further information regarding storage outside refrigerator.
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NICE decisions ▶Patisiran for treating hereditary transthyretin amyloidosis (August 2019) NICE HST10 Patisiran (Onpattro ®) is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy. It is recommended only if the manufacturer provides patisiran according to the commercial arrangement. www.nice.org.uk/guidance/hst10 Scottish Medicines Consortium (SMC) decisions SMC No. SMC2157 The Scottish Medicines Consortium has advised (June 2019) that patisiran (Onpattro ®) is accepted for use within NHS Scotland for the treatment of hereditary transthyretin- mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy. This advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or lower.
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▶Common or very common Arthralgia. dyspepsia. dyspnoea . erythema. increased risk of infection. infusion related reaction. muscle spasms. peripheral oedema. vertigo ▶Frequency not known Vitamin A deficiency
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Manufacturer advises women of childbearing potential should use effective contraception during treatment; if conception is planned, patisiran and vitamin A supplementation should be stopped and vitamin A levels monitored—consult product literature.
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Manufacturer advises avoid unless essential (limited information available)—potential teratogenic risk due to unbalanced vitamin A levels, consult product literature.
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Manufacturer advises avoid—present in milk in animal studies.
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Manufacturer advises caution in moderate or severe impairment (no information available).
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Manufacturer advises caution in severe impairment (no information available).
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Manufacturer advises for intravenous infusion (Onpattro ®), filter through 0.45-micron syringe filter and dilute requisite dose with Sodium Chloride 0.9% to final volume of 200 mL; use a DEHP-free infusion set and give over about 80 minutes through an in-line filter (1.2 micron) at a rate of about 1 mL/minute for the first 15 minutes, then increase to about 3 mL/minute for the remainder of the infusion.
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There can be variation in the licensing of different medicines containing the same drug. Solution for infusion ELECTROLYTES: May contain Sodium ▶Onpattro (Alnylam UK Ltd) A Patisiran (as Patisiran sodium) 2 mg per 1 ml Onpattro 10mg/5ml concentrate for solution for infusion vials | 1 vial P £7,676.47
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300 micrograms/kg every 3 weeks (max. per dose
First-line fragment. Read the complete Indications and Dose section above.
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