Amicus Plummets on 3-Year Delay to U.S. Fabry Drug FDA Filing Plan

Published: Nov 29, 2016

Amicus Plummets on 3-Year Delay to U.S. Fabry Drug FDA Filing Plan November 29, 2016
By Alex Keown, BioSpace.com Breaking News Staff

CRANBURY, N.J. – Shares of Amicus Therapeutics plunged more than 22 percent this morning after the company was forced to lay out plans for another clinical trial for its lead drug candidate migalastat, also known as Galafold in Europe, in order to win regulatory approval. The need for a new trial is expected to delay the company’s plans by at least three years.

Amicus said the new 35-patient trial for a Fabry disease treatment was called for after a meeting with the U.S. Food and Drug Administration. The company said it will collect additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation, which will result in a year-long placebo-controlled "cross-over" study. The new study is expected to begin sometime in 2017, but the company said it will not likely have clinical data until 2019.

Migalastat is designed to bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations, the company said. Fabry disease is a rare genetic disease caused by the lack of an enzyme that allows the body to break down lipids, which are fat-like substances that include oils, waxes and fatty acids. Without the ability to break down the enzyme can lead to kidney problems as well as heart attack or stroke. The disease affects about 10,000 people globally. About half of Fabry disease patients show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting, Amicus said.

"While we believe that the totality of the data from our studies with migalastat support the submission of a new drug application today, we acknowledge the FDA's position that accelerated approval based on kidney GL-3 reduction is not currently an option. We have thus defined a plan to collect additional GI data to support full approval for migalastat that we believe is feasible in a reasonable amount of time and with a high likelihood of success based on positive GI data generated in our previous Phase III Study 011.,” John Crowley, chairman and chief executive officer of Amicus said in a statement. “FDA has been flexible in allowing a crossover design and in our use of established GI endpoints to measure clinical benefit in Fabry patients. We are fully committed to the additional work necessary to move migalastat toward approval in the United States."

Migalastat was approved for use in Europe in May. The drug competes with Shire ’s Replagel in the EU, However, the drug has a different mechanism for action, which means Galafold has the potential to be used alongside Shire’s drug. Sanofi also markets a Fabry disease drug called Fabrazyme. Both Replagel and Fabrazyme act by replacing the enzyme missing in Fabry patients. For patients who still produce some of the enzyme, Galafold aids in getting the enzymes to work better, BioSpace previously reported.

The U.S. market for migalastat is estimated to be approximately 25 percent of the global opportunity, Amicus said this morning.

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