NICE recommends Galafold (migalastat) as a treatment for Fabry disease.-Amicus Therapeutics

The National Institute for Health and Care Excellence (NICE) in draft guidance acknowledged that Fabry disease is a serious condition that has severe effects on the lives of people with the condition, as well as their families and carers. The evaluation committee considered the evidence suggesting that migalastat,from Amicus Therapeutics, has comparable effectiveness to ERT (enzyme replacement therapy), and heard the experiences of the patient and clinical experts.

The committee concluded that the evidence had considerable limitations but, on balance, migalastat was likely to provide similar benefits to ERT. The committee understood that migalastat may have additional benefits because it is taken orally rather than as an infusion. The committee considered that the company’s economic model was broadly appropriate and that the ERG’s exploratory analyses presented a range of possibilities that were consistent with the evidence. It concluded that migalastat provides the additional health benefits of an oral therapy at a lower cost compared with ERT, but that the clinical effectiveness evidence was highly uncertain. It accepted that the value of migalastat compared with ERT had been shown, although it noted that NICE has not evaluated ERT. The committee concluded that the case for national commissioning of migalastat is supported when used as an option for treating Fabry disease. It also concluded that further evidence on both the short- and long-term effectiveness of migalastat and a full evaluation of the costs and benefits of ERT for Fabry disease would be valuable.

Comment: According to Amicus, 855 people in England have Fabry disease, of whom around 140 would be suitable for treatment with Galafold.