1.1 Inotersen is recommended, within its marketing authorisation, as an option for treating stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis. It is recommended only if the company provides inotersen according to the commercial arrangement.
Why the committee made these recommendations
Hereditary transthyretin amyloidosis is a rare condition that severely affects the quality of life of people with the condition, and their families and carers. Current treatment is supportive care.
Clinical trial evidence shows that inotersen slows progression of the disease considerably, although its long-term benefits are uncertain. Some assumptions in the economic modelling are also uncertain, particularly around the utility values and the healthcare costs. Despite the uncertainties, inotersen is likely to provide important clinical benefits for people with hereditary transthyretin amyloidosis and value for money within the context of a highly specialised service. It is therefore recommended for use in the NHS.