|Amicus Therapeutics Shares Fall Hard After The Bell|
|By Staff and Wire Reports|
|Thursday, 20 December 2012 03:59|
The company is developing migalastat HCl in partnership with British drugmaker GlaxoSmithKline plc (GSK.L) to treat Fabry disease, a genetic condition in which a type of fat gets deposited in human tissue, particularly in the kidneys. The disease is caused by the body's inability to produce the enzyme that helps breaks down the fat.
The six-month study found that 13 out of the 32 patients who were put on migalastat HCl reported a reduction of 50 percent or more in the fat deposits in their kidneys, compared to 9 of the 32 in the group that was given a placebo.
"This difference did not achieve statistical significance according to the pre-specified primary endpoint analysis," the company said.
Amicus said it expects to report results from 12 months of treatment in the first half of 2013, after which it plans to meet with the FDA to discuss a U.S. "approval pathway."
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."