Amicus Therapeutics, Inc. Announces Positive Phase 3 Data on Cardiac Endpoints From Fabry Monotherapy Study 011 and Long Term Extension Study
January 08, 2015 at 12:30 pm
Share
Amicus Therapeutics announced additional positive data on an important cardiac endpoint from its first Phase 3 study and the Phase 3 extension of the oral small molecule chaperone migalastat HCl for Fabry disease. Data from the Fabry Registry indicate that the cause of death in patients is from cardiovascular disease1. In Study 011 and Study 041, previously untreated patients with amenable mutations showed a statistically significant decrease in left ventricular mass index (LVMi) following treatment with migalastat for up to 36 months (average of 22 months).
Amicus Therapeutics, Inc. is a biotechnology company, which is focused on discovering, developing, and delivering novel medicines for rare diseases. Its two marketed therapies are Galafold, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti + Opfolda, a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. As an orally administered monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice (GLP) cell-based amenability assay. Pombiliti + Opfolda consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with miglustat that functions as an enzyme stabilizer.
AMICUS THERAPEUTICS, INC. 
