Clinical Trials Resource Center

New Medical Therapies™

Fabry Disease

February 21, 2011

Amicus reported preliminary data from a phase II long-term extension study of Amigal for Fabry Disease. This non-randomized, open-label, uncontrolled, single group extension study initially enrolled 26 subjects with Fabry disease who had completed previous phase II trials. At this time, 15 subjects had been treated with Amigal for more than three years and seven subjects had been treated with migalastat HCl for more than four years. Seventeen subjects are still receiving treatment in the ongoing extension study. Data indicate that estimated glomerular filtration rate (eGFR) has remained stable out to three to four years for all subjects continuing in the extension study. The average annual rate of change in eGFR in subjects identified as responders to Amigal was +1.6 mL/min/1.73m2. Reduced 24-hour urine protein was also observed in multiple subjects identified as responders to Amigal, with a mean 21% and median 34% reduction from baseline.

September 12, 2005

Amicus Therapeutics has announced positive results of a phase I trial of Amigal (migalastat hydrochloride) for the treatment of Fabry disease. Results from the study indicated that the drug was safe and well tolerated, with no serious or drug related adverse events reported. Pharmacokinetic data indicated high oral bioavailability and a favorable overall profile. Preliminary efficacy results were also positive, with statistically significant and dose dependent increases in activity of the target enzyme, alpha-galactosidase A. This open-label study enrolled 16 healthy volunteers. These results formed the basis of Amicus' recently initiated phase II trials of the drug.

December 9, 2002

Transkaryotic Therapies reported negative preliminary results from a phase III trial investigating Replagal (agalsidase alfa), an enzyme replacement for the treatment of Fabry Disease. Results showed that the preliminary data did not reach its primary end-point of renal function, as measured by glomerular filtration rate. The double blind, placebo-controlled study enrolled 80 subjects with Fabry Disease. Subjects were given Replagal (.2 mg/kg) every other week over a 40-minute intravenous infusion in a six-month regimen.

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