Patisiran (Onpattro, Alnylam) is a ribonucleic acid interference agent that suppresses transthyretin (TTR) production by the liver (including abnormal TTR). It is administered once every 3 weeks by intravenous infusion at a dose of 0.3 mg/kg. It has a marketing authorisation in the UK for treating 'hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy'.
The most common adverse reactions listed in the summary of product characteristics for patisiran include peripheral oedema, infusion-related reactions, infections, vertigo, dyspnoea, dyspepsia, erythema, arthralgia and muscle spasms. For full details of adverse reactions and contraindications, see the summary of product characteristics.
The price of patisiran is £7,676.45 per 10‑mg vial (excluding VAT; company submission). The company has a commercial arrangement. This makes patisiran available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.