Chaperone-enzyme replacement therapy product - Amicus/JCR PharmaceuticalsAlternative 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-051
Latest Information Update: 14 Apr 2016
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.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- 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)