NICE (UK) positive for Wainzua (eplontersen) for treating hereditary transthyretin-related amyloidosis – AstraZeneca

NICE (UK): 1.1 Eplontersen is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy. It is only recommended if the company provides eplontersen according to the commercial arrangement. 1.2 Use the least expensive option of the available treatments (including eplontersen and vutrisiran). Take account of administration costs, dosages, price per dose and commercial arrangements. If the least expensive option is unsuitable, people with the condition and their healthcare professional should discuss the advantages and disadvantages of other treatments.

Why these recommendations were made: Usual treatment for hereditary transthyretin-related amyloidosis with stage 1 or 2 polyneuropathy includes vutrisiran. Eplontersen works in a similar way to vutrisiran but people can inject it themselves at home. Eplontersen is used monthly, whereas vutrisiran is used every 3 months. Clinical trial evidence suggests that eplontersen is more effective than placebo at lowering levels of transthyretin in the blood, and increases how long people have before their polyneuropathy gets worse. There are no clinical trials directly comparing eplontersen with vutrisiran. An indirect comparison suggests that eplontersen works as well as vutrisiran. A cost comparison suggests that eplontersen is cost saving compared with vutrisiran. So, it is recommended.