Table 3.
Overview of Fabry Disease-specific treatment options that have been approved or are currently under investigation.
| Treatment Options | ||
|---|---|---|
| Available | - | - |
| Enzyme Replacement Therapy | - | Agalsidase alfa (EMA) |
| - | Agalsidase beta (EMA, FDA) | |
| Chaperone Therapy | - | Migalastat (EMA, FDA) |
| Under Investigation | - | - |
| Substrate Reduction Therapy | - | Lucerastat |
| - | Venglustat | |
| Enzyme Replacement Therapy | - | Pegunigalsidase alfa |
| - | Moss-aGal | |
| Gene Therapy | Ex-vivo | Infusion of autologous hematopoietic stem cells engineered to express α-Gal A (ex-vivo lentiviral gene therapy AVR-RD-01) |
| In-vivo | Recombinant adeno-associated virus vectors (4D-310, ST-920, FLT190) carrying DNA encoding α-Gal A | |
| Solid lipid nanoparticle-based vector carrying DNA encoding α-Gal A | ||
| mRNA-based Therapy | - | Exogenous mRNA via lipid nanoparticles |
α-Gal A: alpha-galactosidase A; EMA: European Medicine Agency; FDA: U.S. Food and Drug Administration.