Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16.

Is this guidance up to date?

Next review: February 2020 or sooner if the prices or availability of enzyme replacement therapies (agalsidase alfa and agalsidase beta) for Fabry disease change.

Any future consideration of the review of migalastat should include formal evaluation of the benefits and costs of agalsidase alfa and agalsidase beta for treating Fabry disease.

Guidance development process

How we develop NICE highly specialised technology guidance

Your responsibility

The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.

All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the Yellow Card Scheme.

Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.

Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible.