migalastat (Galafold)

following a full submission:

migalastat (Galafold®) is accepted for restricted use within NHS Scotland.

Indication under review: long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.

SMC restriction: in males with classic mutations (leucocyte enzyme activity <1%) treatment should commence at diagnosis; in females and those males with later onset mutations with higher levels of leucocyte enzyme activity, treatment should commence when patients experience uncontrolled pain, evidence of renal, cardiac or neurovascular disease, or gastrointestinal symptoms that significantly reduce quality of life.

In an 18-month, randomised, phase III study, migalastat was comparable to enzyme replacement therapy, measured by mean annualised rate of change in glomerular filtration rate.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of migalastat. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.