GALAFOLD (migalastat), chaperone molecule for amenable α-galactosidase mutations

RARE DISEASE - New medicinal product
Opinions on drugs - Posted on Feb 21 2017

Reason for request


Minor improvement in the treatment of Fabry disease, in patients with an amenable mutation .


  • GALAFOLD has Marketing Authorisation in the long-term treatment of patients aged 16 years or older   with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency), and who have an amenable mutation.
  • Its efficacy relative to placebo has been demonstrated in terms of reduction of the mean number of GL-3 inclusions in the renal interstitial capillaries relative to baseline in the study, after 6 months of treatment. However, this is a secondary endpoint, from a subgroup defined a posteriori. This exploratory result should be considered with caution.
  • The results of another study have not made it possible to conclude a difference or equivalence in terms of variation of glomerular filtration rate between GALAFOLD and enzyme replacement therapy.
  • GALAFOLD may be offered as an alternative to enzyme replacement therapy only in patients with an amenable mutation.


Clinical Benefit



Clinical Added Value



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