Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fab... (h Fabry disease that showed that Amigal ...)

Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fabry Disease

Date:8/4/2008

h Fabry disease that showed that Amigal was generally safe and well-tolerated at all doses evaluated. In a majority of patients, treatment with Amigal resulted in increased activity of the enzyme deficient in Fabry patients (alpha-GAL) and a reduction of kidney GL-3 as measured in urine.

In parallel with the regulatory process, 23 of the original 26 patients will continue to be treated with Amigal in a voluntary Phase 2 extension study to monitor long term safety and efficacy and to evaluate additional doses and dose regimens. Data from this extension study are expected to be available by Q1 2009. In addition, Amicus expects to conduct clinical pharmacology studies to support the Phase 3 program.

Amicus is developing Amigal as part of a strategic collaboration with Shire Human Genetic Therapies, Inc., a wholly-owned subsidiary of Shire Limited, to develop and commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire received commercial rights outside of the United States. Amicus retains all U.S. rights.

About Amigal

Amigal (migalastat hydrochloride) is an experimental, oral therapy for the treatment of Fabry disease, a lysosomal storage disorder, which can cause damage to specific areas of the body, including the kidneys, heart, nervous system, and skin. Amigal, a pharmacological chaperone, acts by selectively binding to the misfolded enzyme responsible for Fabry disease, alpha-GAL. After binding to the enzyme, Amigal promotes the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced, and the enzyme can perform its normal, biological function, which is the breakdown of its natural substrate, GL-3.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmac
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