In addition to the binary responder analysis reported above, a pre-specified secondary analysis assessing the absolute percent change in kidney interstitial capillary GL-3 from baseline to month 6 was performed. Taken alone this analysis showed a median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group (p=0.093).
To date, no drug-related serious adverse events have been observed. The most common treatment emergent adverse events occurring in 10% or more of subjects were (migalastat; placebo, respectively): headache (35%; 21%); fatigue (12%; 12%); nausea (12%; 9%); nasopharyngitis, or inflammation of the nose and throat (15%; 6%); and parasthesia, or tingling sensation of the skin (9%; 12%). The 4 dropouts in this portion of the study were deemed by the investigators to be unrelated to study medication.
The 6-month secondary endpoints in Study 011 continue to be analyzed and will be presented at the Lysosomal Disease Network WORLD Symposium ( LDN WORLD), to be held February 12-15, 2013, in Orlando, Florida. Secondary endpoints include urine GL-3 and renal function (iohexol GFR, eGFR and 24-hour urine protein).
John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated "Consistent with our Phase 2 experience, the 6-month results from Study 011 demonstrate notable trends in kidney interstitial capillary GL-3 reduction in favor of migalastat HCl monotherapy compared to placebo. We look forward to announcing additional 6-month results at the WORLD Symposium in February, including a presentation of important secondary and tertiary endpoints in this study. We also anticipate 12-month results from this study in the first half of 2013. Once we have the 12-month data, we intend to meet with FDA to discuss a U.S. approval pathway. We continue to believe that migalastat HCl may become an important treatment option as an oral monotherapy drug for both men and women with Fabry disease who have amenable mutations."