Amicus Therapeutics Inc. Conducts Successful Trial For Fabry Genetic Disorder Drug
By Rebekah Marcarelli r.marcarelli@hngn.com
HNGN
Apr 29, 2014
An Amicus Therapeutics Inc. drug that would reduce abnormal fat that accumulates in cells when a rare genetic disorder is present proved succeeded in trials.
The condition can lead to "heart attack, stroke and kidney failure," Reuters reported.
The company plans to file for U.S. marketing approval in the wake of these results. The drug, dubbed migalastat, could be an effective monotherapy for Fabry disease patients who have endure a year of previous treatment.
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The researchers conducted a 24-month study in which for the first six months participants either received the fat-fighting drug or a placebo. All of the patients were then treated with migalastat for a six-month follow up period and 12-month extension phase, Reuters reported. The drug is administered intravenously.
The drug did not show a significant reduction after six months, but the compant plans to report the results at 12 months.
"It may be that (the drug) needs more time to work, as shown by today's results," Janney Montgomery Scott analyst Kimberly Lee told Reuters.
The drug is also being tested as a combination treatment to be used with other common treatment methods for the condition.
Fabry disease is genetic; it causes a type of fat called "globotriaosylceramide, or GL-3," to build up in cells, especially in the kidney.
This lipid accumulation is accompanied by a reduction in the enzyme α-galactosidase A (α-Gal A), which can lead to "cell damage, leading to pain, hearing loss, kidney failure, heart attacks and strokes," Reuters reported.
Migalastat could combat these devastating effects by binding to the α-Gal A enzyme and helping it to break down the accumulating lipids.
Lee believes the new drug could generate $250 million globally if used as a monotherapy; if the drug is used as a combination therapy it could be worth up to twice as much, Reuters reported.
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