Amicus Therapeutics, Inc. Rockets On Positive Phase 3 Study Data For Rare Disease Drug

Published: Aug 21, 2014

Amicus Therapeutics Rockets On Positive Phase 3 Study Data For Rare Disease Drug Amicus Therapeutics Rockets On Positive Phase 3 Study Data For Rare Disease Drug

August 20, 2014
By Krystle Vermes, Breaking News Staff

Amicus Therapeutics, Inc. , a New Jersey-based biopharmaceutical company, announced today that the oral small molecule chaperone migalastat HCl has shown relatively positive results in its second Phase 3 study. The data from this study was compiled over the course of 18 months.

The second Phase 3 Study, Study 012, compared oral migalastat to enzyme replacement therapies (ERTs) for Fabry disease. Fabry disease is a rare genetic condition that can cause gastrointestinal issues, a lack of sweating and fever. The disease is caused due to a lack of an enzyme called ceramide trihexosidase, which is necessary to metabolize lipids.

During the study, researchers discovered that levels of plasma lyso-Gb3, a critical biomarker of disease, remained low and stable in patients with amenable mutations who switched from ERT to migalastat. After the study, 46 of the 48 patients who participated made the decision to continue with a 12-month treatment extension of migalastat. Today, 45 of the participants still remain on migalastat as their only method of treatment for the disease.

"We believe that this multi-year study unequivocally demonstrates that a Fabry patient on ERT with an amenable mutation can switch safely and effectively from ERT to migalastat to treat their Fabry disease,” said John Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. “Today is a great day for the Fabry community and for Amicus. This study was resoundingly positive and met our pre-defined criteria for success in terms of the co-primary endpoints of kidney function. These results clearly show that migalastat is comparable to ERT in slowing the progression of Fabry disease and continues to demonstrate a favorable safety profile. With every-other-day oral administration and a differentiated mechanism of action, migalastat may offer significant advantages for patients without the need for bi-weekly infusions with ERT.”

A decline in kidney function is often associated with the mortality of patients living with Fabry disease. Through the 18-month study, researchers found that kidney function was comparable to ERT in patients who received migalastat.

"These data mark an exciting day for the Fabry community and validate our long-term commitment to work in partnership with industry toward our goal of multiple treatment options and improved medicines for all people living with Fabry disease," said Jack Johnson, Founder and Executive Director, Fabry Support & Information Group. "We await the regulatory agencies' review of these data, and we are grateful to the many people with Fabry disease, families and volunteers who have committed so much of themselves to help accelerate efforts to bring a more convenient and effective therapy to people living with the disease."

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