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 /
Ref: Amicusrx| Image: MDA
Click here to read the full press release
Tags
This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com