Amicus Therapeutics Reports Positive Phase 3 Data From Fabry Monotherapy Study

(RTTNews) - Amicus Therapeutics, Inc. (FOLD) reported additional positive data from a Phase 3 study or Study011 of the oral small molecule chaperone migalastat HCl in Fabry disease patients with amenable mutations.

The company noted that across all subgroups, patients treated with migalastat compared favorably to natural history on kidney function. Additional GL-3 data further validate assay for identifying patients with amenable mutations, according to the company.

Amicus Therapeutics noted that assessment of kidney function by various measures of glomerular filtration rate or GFR for patients receiving migalastat in Study 011 for at least 18 months and continuing migalastat treatment in Study 041 showed continued stability of kidney function for an average of 32 months. Decline in kidney function is a key cause of morbidity and mortality in patients with Fabry disease.

Measured GFR or mGFR showed stability over 18-24 months in Study 011, but was not collected in Study 041. mGFR was previously reported with topline Study 011 results.