The U.S. Food and Drug Administration (FDA) approved Monday a Bristol-Myers Squibb Co. drug for the treatment of rare and potentially fatal disorders that involve loss of body fat.
The drug, called Myalept (metreleptin for injection), was approved to treat complications of leptin deficiency in patients who have congenital generalized or acquired generalized lipodystrophy, the FDA said.
"Myalept is the first approved therapy indicated for treating the complications associated with congenital or acquired generalized lipodystrophy and provides a needed treatment option for patients with this orphan disease," said Mary Parks, deputy director for the Office of Drug Evaluation II at the FDA's Center for Drug Evaluation and Research.
The drug was evaluated in a clinical study involving 48 people with generalized lipodystrophy. Good results were shown in blood sugar control and reduced blood triglycerides.
According to the FDA, generalized lipodystrophy is a condition involving a lack of fat tissue. Those who have the condition are born with little or no fat tissue. Patients with the condition also tend to lose fat tissue over time.
A serious imbalance in the body results from leptin deficiency, causing fat accumulation in muscles and organs like the liver. Diabetes, pancreatitis and fatty liver disease can all result from the deficiency. Those with lipodystrophy often develop diabetes at a young age and have a difficult time controlling levels of triglycerides, which can cause inflammation of the pancreas.
Myalept is a form of leptin meant to reduce fat accumulation in the organs to create stronger control over blood sugar and high levels of triglycerides: a type of fat in the bloodstream linked to an increased risk of heart disease. Produced by fat tissue, Leptin is a hormone that assists in regulating food intake and other hormones like insulin, the FDA said Tuesday in a news release.
Myalept is marketed by Amylin Pharmaceuticals, a company based in San Diego. Amylin Pharmaceuticals was acquired by the Bristol-Myers Squibb Company in 2012. According to Bristol-Myers, the FDA has asked the company to conduct seven post-marketing studies on the drug. Bristol-Myers co-developed the drug with AstraZeneca.
The use of Myalept has been connected with some serious side effects, including lymphoma and drug resistance. Accordingly, the drug will only be available from a small number of health care professionals certified in prescribing it. Less serious side effects could include headache, low blood sugar, abdominal pain and weight loss. Those with general obesity are not recommended to take the drug. The FDA said that those taking Myalept may develop a resistance to the drug, making it less effective over time and boosting their risk of severe infection.
