1.1 Migalastat is recommended, within its marketing authorisation, as an option for treating Fabry disease in people over 16 years of age with an amenable mutation, only if migalastat is provided with the discount agreed in the patient access scheme, and only if enzyme replacement therapy (ERT) would otherwise be offered. Criteria for starting and stopping ERT for Fabry disease are described in the UK adult Fabry disease standard operating procedures (Hughes et al. 2013). With the discount provided in the patient access scheme, migalastat has a lower total cost than ERT, and potentially provides greater health benefits than ERT.
1.2 The committee noted that there were important limitations and uncertainties in the evidence presented for migalastat, and that NICE has not evaluated ERT (agalsidase alfa and agalsidase beta) for treating Fabry disease. It encourages the company, NHS England and treatment centres to collect more evidence, particularly on the longer-term benefits of migalastat and ERT for treating Fabry disease, which should inform a future evaluation of the costs and benefits of all treatment options for Fabry disease.