Annotation of EMA Label for migalastat and GLA
PharmGKB ID
Summary
The EMA European Public Assessment Report (EPAR) for migalastat (Galafold) states that it is for the treatment of adults and adolescents age 16 years and older with Fabry disease and an "amenable" GLA variant.
Annotation
Excerpts from the migalastat (Galafold) EPAR:
Galafold is indicated for 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...
Certain GLA mutations can result in the production of abnormally folded and unstable mutant forms of α-Gal A. Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of α-Gal A, the genotypes of which are referred to as amenable mutations...
The GLA mutations amenable and not amenable to treatment with Galafold are listed in Table 2 and Table 3 respectively below. The GLA mutations are also accessible by health care providers at www.galafoldamenabilitytable.com.
For the complete EPAR text with sections containing pharmacogenetic information highlighted, see the migalastat EPAR PDF
*Disclaimer: The contents of this page have not been endorsed by the EMA and are the sole responsibility of PharmGKB.
Evidence for Clinical Annotations
This annotation has been used as evidence for the following clinical annotations.