Amicus Therapeutics (FOLD) says that top-line data and statistical analysis from the clinical trail (Study 012) evaluating its oral small molecule pharmacological charperone migalastat HCL as a monotherapy for Fabry disease compared to standard-of-care enzyme replacement therapies (ERT) Fabrazyme and Replagal should be available next quarter. The 18-month primary treatment period was completed in Q2.
The primary endpoint is renal function at 18 months. Specifically, the co-primary outcome measures are the mean annualized changes in measured (iohexol) glomerular filtration rate (mGFR) and estimated GFR (eGFR) for migalastat compared to ERT. The numerical targets are a 50% overlap in the confidence intervals between migalastat and ERT and whether the mean annualized changes in the migalastat cohort are within 2.2 ml/min/1.73 m2/yr of patients receiving ERT.
The company intends to eventually file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) contingent on the successful outcome of Study 012.