The company, whose shares rose as much as 36
percent, said it would file for U.S. marketing approval based on
these results and those from an European trial, expected in the
The late-stage trial evaluated the drug, migalastat, as a form of
monotherapy for certain Fabry disease patients after 12 months of
The 24-month study began with a 6-month period where patients
received either the drug or a placebo. All patients were then
treated with migalastat for a 6-month follow-up period and a
subsequent 12-month extension phase.
After the drug failed to show statistically significant reduction in
kidney lipid levels at 6 months, Amicus said it would report
12-month efficacy and safety data to support its marketing
"It may be that (the drug) needs more time to work, as shown by
today's results," Janney Montgomery Scott analyst Kimberly Lee said,
adding that the European trial data would provide further evidence
of whether the drug needed a longer duration to be effective.
Cowen & Co analyst Edward Nash said migalastat could satisfy the
"significant need for an oral therapy for Fabry disease".
Sanofi SA's Fabrazyme, the first FDA-approved Fabry treatment, is
Lee said she expected migalastat's use as a monotherapy could
represent a $250 million global market opportunity and be worth up
to double that size as a combination therapy.
Amicus is also testing migalastat in combination with the current
standard-of-care for the disease.
GlaxoSmithKline returned the rights to migalastat to Amicus in
[to top of second column]
Fabry disease is an inherited, potentially fatal disorder
characterized by the buildup of a particular type of fat — most
notably in the kidneys — called globotriaosylceramide, or GL-3, in
the body's cells.
This progressive lipid accumulation, caused by the deficiency of the
enzyme α-galactosidase A (α-Gal A), results in cell damage, leading
to pain, hearing loss, kidney failure, heart attacks and strokes. As
a monotherapy, migalastat works by binding to the α-Gal A enzyme,
made in the patient's body, helping it break down the lipids. The
Cranbury, New Jersey-based company's stock was up about 22 percent
at $2.25 in afternoon trading.
(Reporting by Natalie Grover in Bangalore;
editing by Don Sebastian
and Sriraj Kalluvila)
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