Amicus Therapeutics Releases Positive Trial Results For Experimental Fabry Disease Drug

Amicus Therapeutics issued a press release announcing that its experimental Fabry disease drug, migalastat, has performed well in its late stage clinical trial. The 24-month double blind study proceeded with a 6-month placebo-controlled treatment period and was followed by another six months, during which patients were treated with migalastat for a combined 18-month open-label follow-up period and extension phase.

The results show that patients who stayed on migalastat for 12 months were able to see a reduction in kidney interstitial capillary GL-3 compared to those on placebo. Patients treated also maintained stability in kidney function over the same period. Patients in the study on the placebo who switched to migalastat in month 6 also saw a significant reduction in kidney interstitial capillary GL-3 by month 12.

John F. Crowley, the chairman and CEO of Amicus Therapeutics, said that the data was extremely promising. “Today is a great day for Amicus and the Fabry community. We are pleased to report that the 12 and 24 month results from Study 011 have met our pre-defined criteria for success in terms of substrate reduction at 12 months, as well as clinical measures of kidney function maintained out to 24 months.,” said Crowley. “We believe these data provide important validation that a small-molecule chaperone can restore the function of a patient's own enzyme in patients with amenable mutations… these results demonstrate the power of personalized medicine in rare diseases and offer the prospect of a new treatment option that differs from traditional enzyme replacement therapy.”

The company has also stated that it plans to work with regulatory authorities to determine the quickest way to go about bringing the drug to market.

Fabry disease is inherited. It results from a buildup of fat called globotriaosylceramide in the body’s cells. The symptoms include pain in the hands and feet, small, dark red spots on the skin, a reduced ability to sweat, ringing in the ears, and cloudiness in the front part of the eye. Over time, Fabry disease can cause heart attacks, strokes, and serious kidney damage.