Orphan status for Gaucher disease drug; Fabry disease clinical trials

In February 2006 the U.S. Food and Drug Administration (FDA) approved orphan drug status for AT2101 (Amicus Therapeutics) for the treatment of Gaucher disease, a lysosomal storage disorder resulting from deficiency of the enzyme glucocerebrosidase. AT2101 is an oral drug which helps restore the proper structure and cellular movement of glucocerebrosidase. Clinical studies of the drug are expected to begin during the first half of 2006.

Amicus Therapeutics is also conducting Phase II clinical trials for the drug AT1001 (migalastat hydrochloride) for treatment of another storage disorder, Fabry disease.