(Adds company statement)
Dec 11 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 a condition known as generalized
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
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.
(Reporting by Bill Berkrot; editing by Matthew Lewis, Dan
Grebler and Diane Craft)