3 The technology

3.1 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'.

3.2 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.

3.3 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.

  • National Institute for Health and Care Excellence (NICE)