Apabetalone (RVX-208) is an investigational oral medication being developed for the treatment of several conditions, including Fabry disease. Apabetalone is a selective bromodomain and extra-terminal inhibitor. BET bromodomain inhibition is a new approach that can regulate disease-causing genes. It is currently in Phase 1/2 trials for the treatment of Fabry.
Gene therapy for Fabry disease is designed to introduce a healthy copy of the GLA gene inside cells to restore normal levels of the alpha-galactosidase A enzyme. Several potential gene therapies are now being investigated for the treatment of Fabry disease.
Substrate Reduction Therapy
Substrate reduction therapy is an investigational treatment method for Fabry disease and other lysosomal storage disorders. Substrate reduction therapy takes its name from the fact that it reduces the amounts of the substrate of a certain enzyme. In this way, the therapy ensures that lack of this enzyme in Fabry disease is no longer a problem.
PRX-102 (pegunigalsidase alfa) is an investigational enzyme replacement therapy (ERT) for Fabry disease. The potential treatment is designed to reduce the number of required enzyme infusions to once a month, rather than every two weeks with existing ERTs. PRX-102 is currently under review for approval by the U.S. Food and Drug Administration.