Amicus Therapeutics drug Migalastat found effective in trial to treat Fabry disease

Amicus Therapeutics Inc reports significant reductions in abnormal fat that accumulates in some cells of patients with a rare genetic disorder that can cause kidney failure, stroke, and heart attack.

by John Tyburski

Copyright © Daily Digest News, KPR Media, LLC. All rights reserved.

First published at Daily Digest News on April 29, 2014

According to a report by Reuters on Wednesday, New Jersey-based Amicus Therapeutics Inc. announced that in a 24-month trial, its new drug migalastat was effective at reducing abnormal fat accumulation in kidney cells compared with placebo. The drug is being considered alone and in combination therapy for treating Fabry disease, a congenital disorder that can be debilitating and even fatal.

Fabry disease involves a deficiency in an enzyme important in lipid metabolism called α-galactosidase A, or α-Gal A. When this enzyme is not functioning, a fat called globotriaosylceramide, or GL-3, accumulates to abnormal levels in some cells of the body, particularly cells of the kidney. This accumulation of GL-3 eventually leads to cell injury and death. Migalastat, delivered orally, boosts the performance of α-Gal A when it binds to the enzyme.

The study of migalastat in Fabry patients lasted two years in which patients during the first six months took either the drug or placebo. All patients were then treated with the drug for another six months followed by a 12-month extension phase. Scientists did not observe a significant reduction in GL-3 at six months but did at 12 months, suggesting that the drug may need up to a year to exert its beneficial effects. The report did not disclose any side effects or adverse outcomes.

Results of the pending European trial are anticipated to confirm whether the drug works and needs such a long treatment period. Meanwhile, the announcement was accompanied by a 22 percent increase in the company’s stock price and trading at $2.25 per share at last report.

In addition to testing the drug on its own, Amicus Therapeutics is studying the effectiveness of migalastat combined with the current standard-of-care for Fabry disease. The company plans to file for marketing approval in the U.S. using these results and anticipated results from a European trial set to end later this year.