Amigal migalastat: Preliminary Phase II data

Data from the 6 patients in the open-label Phase II Study 013 showed a positive drug-drug interaction between 150 mg Amigal and Fabrazyme agalsidase beta. In 4 patients receiving 0.5 mg/kg Fabrazyme every 2 weeks, co-administration with Amigal led to a 2- to 4.2-fold increase in plasma active enzyme as measured by total area under the curve (AUC) vs. ERT alone. In skin biopsies from 3 of the patients, the combination led to a 1.1- to 3.9-fold increase in active enzyme at day 2 but no increase at day 7 vs. ERT alone.

In 2 patients receiving 1 mg/kg Fabrazyme every 4 weeks, co-administration with Amigal led to a 1.6 and 2.2-fold increase in plasma active enzyme as measured by total AUC vs. Fabrazyme alone. In skin biopsies, active enzyme increased 1.6- and 2.1-fold at day 2 and 1.2 and 1.7-fold at day 7 vs. Fabrazyme alone. Data will be presented at the Lysosomal Disease Network meeting in San Diego in February. ...