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

RARE DISEASE - New medicinal product

Opinions on drugs - Posted on Feb 21 2017

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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.

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