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ARTICLE | Clinical News

Migalastat: Additional Phase III data

February 18, 2013 8:00 AM UTC

Amicus reported data from a post hoc analysis of the modified intent-to-treat (mITT) population (n=60) from the double-blind, placebo-controlled, international Phase III FACETS (Study 011) trial in 67 patients with Fabry's disease evaluating 150 mg oral Amigal given every other day. In patients with a higher baseline disease burden (n=25), defined as >=0.3 GL-3 inclusions per kidney interstitial capillary, 64% of patients receiving Amigal achieved a >=50% reduction in kidney interstitial capillary GL-3 levels as measured in kidney biopsies from baseline to 6 months, the primary endpoint, vs. 14% for placebo. In patients with a lower baseline disease burden (n=35), 32% of patients receiving Amigal achieved the endpoint vs. 44% for placebo. The company did not disclose p-values.

Additionally, Amigal led to a median reduction in urine GL-3 levels from baseline to 6 months of 12% vs. 17% for placebo in the mITT population. However, the company said that any potential treatment effect on urine GL-3 levels could not be determined due to unexpected variability in pre-treatment urine GL-3 data. Amigal also led to a mean increase in estimated glomerular filtration rate (eGFR) of 2.7 mL/min/1.73 m2 vs. a mean decrease of 2.4 mL/min/1.73 m2 for placebo. No clinically meaningful changes in proteinuria were observed. The trial enrolled Fabry's disease patients who had genetic mutations amenable to chaperone monotherapy in a cell-based assay. Data were presented at the Lysosomal Disease Network World Symposium in Orlando. Amicus expects 12-month data from patients who continued to receive Amigal or crossed over to receive Amigal in 2Q13. Amicus said it looks forward to reporting the 12-month results "to add to the entirety of the data that FDA has indicated would support a potential U.S. conditional approval." The company declined to disclose details, but said it plans to meet with FDA in mid-2013 to discuss an approval pathway. ...