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Amicus to Seek Accelerated US and European Approval of its Investigational Fabry Disease Drug

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Amicus Therapeutics announced its plans to file for accelerated approval of its investigational drug for a rare, inherited disease, following meetings with regulators in the US and Europe.

The company said that it has recently met with regulatory authorities in Europe and the US to discuss the approval pathways for the oral small molecule pharmacological chaperone migalastat HCI as a precision medicine monotherapy for Fabry patients who have amenable genetic mutations.

Amicus said that it plans to submit file papers for approval of migalastat in Europe in the second quarter of 2015. And based on the company’s meeting with US regulators, it plans to submit an application to the US Food and Drug Administration (FDA) in the second half of 2015.

Fabry disease is an inherited lysosomal storage disorder that affects the way certain chemicals are processed in the body. In patients with Fabry disease, cells store up a fatty substance called globotriaosylceramide (GL-3).  The disease involves potentially life-threatening complications, such as progressive kidney damage, heart attack, and stroke. Some affected patients have milder forms of the disorder that appear later in life and affect only the heart or kidneys.

Currently, Fabry disease patients are limited to treatments that require bi-weekly IV infusions, such as Sanofi’s Fabrazyme or Shire’s Replagal. If approved, Amicus’ migalastat would be the first oral treatment available for these patients. As a pill, the drug would be more convenient for patients, giving it a potentially significant competitive advantage over current therapies.

Amicus pulled a previous FDA application for the drug after migalastat missed its goal in its first Phase III study in 2012. After evaluating the study data, the company found that patients with certain mutations were responding better. Based on results from mutations tested in the GLP HEK assay, a test to measure the criteria for amenability with more quality control and rigor, Amicus believes that approximately 30 to 50 percent of the Fabry population have mutations that are amenable to migalastat.

“We are very pleased with these recent regulatory interactions. These regulatory discussions demonstrate that health officials in both the U.S. and EU recognize the continued life threatening medical challenges that people living with Fabry face each day. It has been a stellar example of industry and regulators working together to evaluate data and to determine the best and fastest ways in which to bring novel therapies to people in need, especially in such a rare and devastating disease like Fabry. The Amicus team is working tirelessly to submit the highest quality submission to the EU in the netx quarter and to the United States in the second half of this year. This is another great step forward for people living with Fabry and the potential for a new precision medicine to treat their disease,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc.

Source: Amicus Therapeutics, Inc.

Last updated: 3/20/15; 2:10pm EST

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