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.
Showing posts with label fat. Show all posts
Showing posts with label fat. Show all posts
Tuesday, April 29, 2014
Friday, July 2, 2010
When Fat Makes You Healthy: Subcutaneous Fat May Produce Beneficial Hormones
When Fat Makes You Healthy: Subcutaneous Fat May Produce Beneficial Hormones
May 22, 2008
Diabetes Health
It has long been known that type 2 diabetes is linked to obesity, particularly fat inside the belly. Now, researchers at the Joslin Diabetes Center have found that fat from other areas of the body can actually reduce insulin resistance and improve insulin sensitivity.
In a study published in the May issue of Cell Metabolism, a team lead by C. Ronald Kahn, M.D., found that subcutaneous fat—fat below the skin (usually in the hips and thighs)—is associated with reduced insulin levels and improved insulin sensitivity.
Kahn says that fat in the belly increases the risk of diabetes and mortality and it has been suggested that obesity in subcutaneous areas, often the hips and thighs, might decrease such risks.
His study concludes that fat under the skin is intrinsically different from visceral fat (belly fat or fat packed in between organs) and may produce substances that can improve glucose metabolism.
“The surprising thing was that it wasn’t where the fat was located,” Kahn said. “It was the kind of fat that was the most important variable. Even more surprising, it wasn’t that belly fat was exerting negative effects, but that fat under the skin was producing a good effect. Animals with more of this kind of fat didn’t gain as much weight as they aged, had better insulin sensitivity, lower insulin levels, and were improved all around.”
In addition, Kahn noted that a class of diabetes drugs called thiazoladinediones (ACTOS, Avandia) may cause patients to gain weight in the subcutaneous area, yet also improve insulin sensitivity.
Kahn said it is possible that fat underneath the skin may be producing certain hormones, known as adipokines, that produce beneficial effects on metabolism. These effects may offset the negative effects produced by belly fat.
May 22, 2008
Diabetes Health
It has long been known that type 2 diabetes is linked to obesity, particularly fat inside the belly. Now, researchers at the Joslin Diabetes Center have found that fat from other areas of the body can actually reduce insulin resistance and improve insulin sensitivity.
In a study published in the May issue of Cell Metabolism, a team lead by C. Ronald Kahn, M.D., found that subcutaneous fat—fat below the skin (usually in the hips and thighs)—is associated with reduced insulin levels and improved insulin sensitivity.
Kahn says that fat in the belly increases the risk of diabetes and mortality and it has been suggested that obesity in subcutaneous areas, often the hips and thighs, might decrease such risks.
His study concludes that fat under the skin is intrinsically different from visceral fat (belly fat or fat packed in between organs) and may produce substances that can improve glucose metabolism.
“The surprising thing was that it wasn’t where the fat was located,” Kahn said. “It was the kind of fat that was the most important variable. Even more surprising, it wasn’t that belly fat was exerting negative effects, but that fat under the skin was producing a good effect. Animals with more of this kind of fat didn’t gain as much weight as they aged, had better insulin sensitivity, lower insulin levels, and were improved all around.”
In addition, Kahn noted that a class of diabetes drugs called thiazoladinediones (ACTOS, Avandia) may cause patients to gain weight in the subcutaneous area, yet also improve insulin sensitivity.
Kahn said it is possible that fat underneath the skin may be producing certain hormones, known as adipokines, that produce beneficial effects on metabolism. These effects may offset the negative effects produced by belly fat.
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