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News from Medical News Today Mar. 17,
2006
Amicus Therapeutics Completes Phase
2 Enrollment Of Amigal(TM) For Fabry Disease
Main Category: Biology / Biochemistry News
Article Date: 17 Mar 2007 - 0:00 PDT
Amicus Therapeutics, a biopharmaceutical company developing
small-molecule, orally administered pharmacological chaperones for
the treatment of human genetic diseases, announced today that
enrollment has been completed for all ongoing Phase 2 clinical
trials of Amigal(TM) (migalastat hydrochloride, AT1001). Amigal is
in development for the treatment of
Fabry disease.
The four open-label, multi-national Phase 2 trials are examining
various dose levels and frequencies of Amigal in men and women with
Fabry disease. The primary objective of the studies is to evaluate
the safety and tolerability of treatment with Amigal. The secondary
objective is to evaluate certain pharmacodynamic measures of
treatment, including effects on alpha-galactosidase A (alpha-GAL)
and globotriaosylceramide (GL-3) levels in various cells and tissues
of disease. An additional objective is the preliminary assessment of
cardiac, renal and central nervous system function. The results of
these clinical trials are expected to be available by the end of
2007.
Amigal is designed to selectively bind to and stabilize alpha-GAL,
the enzyme deficient in Fabry disease. This deficiency leads to
lysosomal accumulation of GL-3, which is believed to cause the
various symptoms of Fabry disease. Amigal facilitates proper
trafficking of the enzyme to the lysosomes, the compartments in the
cell where it is needed to break down GL-3.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by inherited
genetic mutations in the GLA gene, which result in deficient
activity of the enzyme alpha-galactosidase A (alpha-GAL). Deficient
alpha-GAL activity leads to lysosomal accumulation of
globotriaosylceramide (GL-3), which is believed to cause the various
symptoms of Fabry disease, including pain, kidney failure and
increased risk of heart attack and stroke. Fabry disease is
estimated to affect approximately 5,000 to 10,000 people in the
developed world, but recent evidence suggests that the disease may
be significantly underdiagnosed. The U.S. Food and Drug
Administration's Office of Orphan Products Development has granted
orphan designation for Amigal in the United States, and the European
Commission has designated Amigal as an orphan medicinal product in
the European Union.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel,
oral therapeutics known as pharmacological chaperones for the
treatment of a range of human genetic diseases. Pharmacological
chaperone technology involves the use of small molecules that
selectively bind to and stabilize proteins in cells, leading to
improved protein folding and trafficking, and increased activity.
Amicus is initially targeting lysosomal storage disorders, which are
severe, chronic genetic diseases with unmet medical needs. Amicus is
currently conducting Phase 2 clinical trials for its two lead
compounds, Amigal(TM) for Fabry disease, and AT2101 for Gaucher
disease. The company is currently conducting Phase 1 trials with
AT2220 for the treatment of Pompe disease.
Amicus Therapeutics
http://www.amicustherapeutics.com /
http://www.medicalnewstoday.com/medicalnews.php?newsid=65428&nfid=rssfeeds
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