A drug has been approved for the treatment of a rare body fat disorder.
The treatment will act as a replacement therapy for patients suffering from leptin hormone deficiency complications, Reuters reported. These complications are a result of congenital or acquired generalized lipodystrophy.
These patients generally suffer from a loss of fat tissue under the skin that causes a reduction in leptin. This can lead to fat accumulation in organs such like the liver, putting the patient at risk of condition such as "diabetes, pancreatitis and fatty liver disease," Reuters reported.
The drug Myalept, from Amylin Pharmacies, is a form of leptin that helps to replace the lost hormone and helps to reduce the amount of fat accumulated in the organs as well as control blood sugar triglyceride levels (fat in the bloodstream that has been linked to heart disease).
This is the first drug approved to treat this condition, MedScape reported.
An FDA advisory committee voted 11 to one to approve the drug for use in children and adults in December 2013. TheEndocrinologic and Metabolic Drugs Advisory Committee voted 10 to two against the use of the drug for "metabolic disorders associated with partial lipodystrophy, including hypertriglyceridemia and/or diabetes mellitus inadequately controlled on a current therapy and/or evidence of hepatic steatosis," MedScape reported.
The treatment is not approved for "HIV-related lipodystrophy or in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy. And it is contraindicated in patients with general obesity," MedScape reported.
To start the drug will only be available through a risk evaluation and mitigation strategy (REMS) program; this will require special certification from the prescriber and pharmacy.
The panel members said they believed the REMS monitoring would find the benefits of the drug will outweigh the risks and side effects.
