Approach to the selection of Fabry-specific therapy in patients with Fabry disease

GLA: galactosidase alpha; eGFR: estimated glomerular filtration rate; ERT: enzyme replacement therapy.

* Indications vary depending on the patient's clinical phenotype. Refer to UpToDate content on indications to initiate Fabry-specific therapy in patients with Fabry disease.

¶ GLA gene variants that are likely to result in a substantial increase in enzyme level may be candidates for migalastat monotherapy instead of ERT. An online tool to determine whether a specific patient's GLA gene variant is amenable to migalastat therapy is available at https://www.galafoldamenabilitytable.us.

Δ Refer to UpToDate content on monitoring for disease progression in patients with Fabry disease.

◊ Migalastat has not yet been approved in the United States for use in children <18 years of age but is approved in the European Union for children ≥12 years old. In addition, migalastat should not be used in patients with an eGFR <30 mL/min/1.73 m², as such patients were excluded from the clinical trials evaluating its efficacy and safety.

§ Available formulations of ERT include agalsidase alfa, agalsidase beta, and pegunigalsidase alfa (for patients >18 years old). Refer to UpToDate content for information regarding dosing and administration of ERT.