Amicus' Amigal improves symptoms among Fabry disease patients in trial

CRANBURY, N.J. — Amicus Therapeutics received good results from its mid-stage trial of a drug for a rare, genetic disorder, the drug maker said Wednesday.

Amicus announced results of a phase-2 extension study of the investigational drug Amigal (migalastat hydrochloride) designed to improve kidney function in patients with the enzyme disorder Fabry disease. The study — an extension of the original, three- to six-month phase-2 study — used two criteria to measure kidney function, protein in the urine during a 24-hour period and a commonly used measure called estimated glomerular filtration rate, or eGFR. Twenty-six patients participated in the original study, while 23 enrolled in the extension study.

The company said preliminary data indicated that eGFR was stable after three to four years of treatment with Amigal, while protein levels in the urine also were reduced among patients in the extension study.

The company plans to present study results at the Lysosomal Disease Network WORLD Symposium in Las Vegas this week.

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