Summary

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• Galafold (migalastat) is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• The systematic review found that assessing the effectiveness of disease-specific therapies, such as enzyme replacement therapy (ERT) and migalastat, on cardiovascular manifestations in Fabry disease was challenging due to significant heterogeneity in study designs and outcome measurements across 72 included studies.
• In the context of gastrointestinal (GI) manifestations in Fabry disease, the review identified that while enzyme replacement therapy and migalastat were commonly used treatments, not all patients responded to enzyme replacement therapy, often necessitating symptomatic relief for conditions like abdominal pain and bowel movement disorders.
• Specific effectiveness outcomes for migalastat compared to enzyme replacement therapy in both cardiovascular and GI manifestations were not clearly detailed, with no distinct population subgroups identified or discussed in relation to these outcomes.
• There is no safety information available in the reviewed studies regarding the safety profile of migalastat for both cardiovascular and gastrointestinal manifestations in Fabry disease patients.