Drug Profile
Chaperone-enzyme replacement therapy product - Amicus/JCR Pharmaceuticals
Alternative Names: Alpha-galactosidase A/migalastat; Alpha-galactosidase/migalastat; AT-B100; Chaperone-ERT product - Amicus/JCR; JR-051/migalastat; Migalastat/alpha galactosidase; Migalastat/alpha galactosidase A; Migalastat/JR-051Latest Information Update: 14 Apr 2016
Price :
$50
*
At a glance
- Originator Amicus Therapeutics
- Developer Amicus Therapeutics; JCR Pharmaceuticals
- Class Enzymes; Pharmacological chaperones; Piperidines; Recombinant proteins; Small molecules
- Mechanism of Action Alpha-galactosidase stimulants; Protein folding stimulants
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Discontinued Fabry's disease
Most Recent Events
- 17 Jul 2012 Preclinical trials in Fabry's disease in USA (Parenteral)