Amicus’s Galafold (migalastat) Receives FDA Approval for Treating Fabry Disease after 15 yrs

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

Click here to read full press release/ article | Ref: Amicusrx| Image:  MDA