Marc Dunoyer, Global Head of GSK Rare Diseases added, "GSK and Amicus are committed to advancing migalastat HCl as a monotherapy in Fabry patients with amenable mutations. While these 6-month data are encouraging, there is additional work to be done. We continue to analyze the 6-month results and look forward to receiving the 12-month results from this study. In addition the results of Study 012, our second Phase 3 Fabry monotherapy study, will add to the totality of our data and give us a more complete picture of the clinical effect of migalastat HCl. This study, an 18-month comparison of migalastat to ERT, with iohexol GFR as the primary endpoint, is fully recruited and due to report in 2014."The 6-month primary treatment period in Study 011 was completed in June 2012 in 63 out of 67 randomized patients. All 63 of these patients entered the 6-month open-label follow-up period in Study 011, to continue to receive migalastat HCl or to switch from placebo to migalastat HCl. In December 2012, a total of 59 patients completed this treatment period and received an additional kidney biopsy at month 12. In addition, 57 out of 59 patients who completed Study 011 continue to receive migalastat HCl in both ongoing open-label extension studies. The results from the 6-12 month period of study 011 are expected in the first half of 2013 and will include 12-month data in the migalastat HCl group and 6-month data in the placebo crossover group.
Amicus Therapeutics And GlaxoSmithKline Announce Top Line 6-Month Primary Treatment Period Results From First Phase 3 Fabry Monotherapy Study
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