Shots:  • Galafold is a first precision and oral drug (Q2D) for 348 amenable galactosidase alpha gene (GLA) variants to treat Fabry in U.S.  • The approval was under Subpart H Accelerated Approval pathway based on reduction of KIC GL-3 substrate  • Approval is based on Ph 3 FACETS study (Study 011) in treatment-naive patients showing efficacy and reducing the amount of disease substrate in kidney-Amicus continues to study Galafold in Ph 4 /

Ref: Amicusrx| Image:  MDA