Indications and Purposes
| 1 Indications And Usage Galafold 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 [See Dosage And Administration ( 2.1 ) And Clinical Pharmacology ( 12.1 )] . This Indication Is Approved Under Accelerated Approval Based On Reduction In Kidney Interstitial Capillary Cell Globotriaosylceramide (Kic Gl-3) Substrate [See Clinical Studies ( 14 )] . Continued Approval For This Indication May Be Contingent Upon Verification And Description Of Clinical Benefit In Confirmatory Trials. Galafold Is An Alpha-Galactosidase A (Alpha-Gal A) Pharmacological Chaperone 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. ( 1 , 12.1 ) This Indication Is Approved Under Accelerated Approval Based On Reduction In Kidney Interstitial Capillary Cell Globotriaosylceramide (Kic Gl-3) Substrate. Continued Approval For This Indication May Be Contingent Upon Verification And Description Of Clinical Benefit In Confirmatory Trials. ( 1 ) |
All Formulated Excipients (0 Total)
None
Active Ingredients ( 1 Total)
| Name | Structure | ZINC ID(s) |
|---|---|---|
| 1. Migalastat Hydrochloride |
|
ZINC1636704 |