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Reuters Health Information (2013-12-11): FDA panel backs drug for generalized lipodystrophy

Regulatory

FDA panel backs drug for generalized lipodystrophy

Last Updated: 2013-12-11 18:51:22 -0500 (Reuters Health)

(Reuters) - An advisory panel of medical experts convened by the U.S. Food and Drug Administration on Wednesday said that Bristol-Myers Squibb Co had provided adequate evidence of the benefits of an experimental drug to treat rare and potentially fatal disorders involving loss of body fat.

The panel voted 11-1 that the benefits of the drug metreleptin outweigh the risks for the treatment of children and adults suffering from generalized lipodystrophy.

Only a few thousand people worldwide are believed to have the disorders, in which fat builds up in the blood and organs such as liver and muscle, and can lead to diabetes, pancreatitis and fatty liver disease. There are currently no approved drugs to treat the underlying causes of the disease, including deficiencies of the human hormone leptin that occur with loss of fat tissue under the skin.

However, by a 10-2 vote, the panel felt the risks of the medicine were too high to recommend it for metabolic disorders associated with partial lipodystrophy, such as diabetes and high triglycerides inadequately controlled by a current therapy.

The FDA typically follows the advice of its expert panels but is under no obligation to do so.

Bristol-Myers and AstraZeneca, which are co-developing the drug, said in a statement that they are confident in the safety and efficacy data provided to the agency for both indications discussed by the panel.

"We remain committed to pursuing metreleptin for treatment in patients with metabolic disorders associated with partial lipodystrophy," the companies said.

With both votes, the panel was instructed to consider its decision taking into account a proposed Risk Evaluation and Mitigation Strategy that would be a requirement of FDA approval. REMS programs typically include registries of patients prescribed the medicine, strong warnings of the risks and other safeguards.

Metreleptin is a form of leptin meant to reduce accumulation of fat in organs to better control blood sugar and high levels of triglycerides - a type of fat in the bloodstream associated with increased risk of heart disease.

The drug has been tested since 2000 by the U.S. National Institutes of Health. Bristol-Myers acquired the commercial rights to the medicine and partnered with AstraZeneca. It is currently awaiting a U.S. approval decision.

 
 
 
 
                 
 
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