Positive data from study of Amigal in Fabry Disease-Amicus Therapeutics

A new study shows Fabry disease patients treated with Amicus Therapeutics' Amigal (migalastat) exhibited improvements in the size of their hearts and fewer disease-related events compared to similar patients treated with currently approved injectable therapies. The new data compliments results from a successful Phase III study announced in August 2014. Fabry patients who were switched to migalastat, a pill taken every other day, from Fabrazyme or Replagal showed a statistically significant decrease in left ventricular mass index (LVMi) compared to patients who remained on the injectable therapies after 18 months.

Treatment with migalastat was also associated with a lower incidence of Fabry-related clinical events compared to Fabrazyme or Replagal by a margin of 29% to 44%, although the difference was not statistically significant. On all the components of the Fabry-associated clinical events score -- cardiac, kidney and central nervous system -- migalastat-treated patients reported fewer events than the injectable therapies. Results from the Phase III study were presented at the American Society of Nephrology annual meeting.