Amicus Therapeutics has completed enrollment into Phase II clinical trials of Amigal, which is in development for the treatment of Fabry disease.
Four open-label, multinational, Phase II trials are examining various dose levels and frequencies of Amigal (migalastat hydrochloride) in men and women with Fabry disease. The primary objective of the studies is to evaluate the safety and tolerability of treatment with Amigal. The secondary objective is to evaluate certain pharmacodynamic measures of treatment, including effects on alpha-galactosidase A (alpha-GAL) and globotriaosylceramide (GL-3) levels in various cells and tissues of disease. An additional objective is the preliminary assessment of cardiac, renal and central nervous system function. The results of these clinical trials are expected to be available by the end of 2007.
Amigal is designed to selectively bind to and stabilize alpha-GAL, the enzyme deficient in Fabry disease. This deficiency leads to lysosomal accumulation of GL-3, which is believed to cause the various symptoms of Fabry disease. Amigal facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down GL-3.
Fabry disease is a lysosomal storage disorder caused by inherited genetic mutations that causes pain, kidney failure and increased risk of heart attack and stroke. The FDA and the European Commission have both granted orphan designation to Amigal.