FDA Approves Myalept for Lipodystrophy Treatment

The US Food and Drug Administration (FDA) approved Myalept, a drug form of the hormone leptin, for the treatment of lipodystrophy. The drug regulates metabolism and curbs the appetite of affected patients, according to a statement provided by UT Southwestern Medical Center.

Lipodystrophy, a rare disorder affecting 300 to 500 people worldwide, is defined by insufficient fat tissue often resulting in insulin resistance. While most patients are affected in childhood, the disease also leads to diabetes or hypertriglyceridemia that can inflame the pancreas. Traditional treatment for lipodystrophy included high-dose insulin plus triglyceride- or lipid-lowering medications.

To ensure Myalept’s safety, its effects were explored in a single-arm study, the FDA explained in a statement. For their study, 48 patients with diabetes mellitus, hypertriglyceridemia and/or elevated levels of fasting insulin who also had congenital or acquired generalized lipodystrophy were observed under treatment of Myalept. Under the trial, patients experienced reductions in HbA1c, a measure of blood sugar control, fasting glucose, and triglycerides. The FDA is requiring 7 additional studies to understand the risks and long term effects of Myalept.

However, there were some observed limitations to leptin therapy. The prevalence of certain antibodies could neutralize the effects of leptin, decreasing the usefulness of Myalept. Side effects of Myalept include hypoglycemia, headache, decreased weight, and abdominal pain.

Abhimanyu Garg, MD, who has lipodystrophy treatments for 3 decades and assisted with the trial, believes leptin therapy provides relief to lipodystrophy patients.

“Many lipodystrophy patients have benefited from leptin therapy,” Garg said. “While it is not a cure, leptin does help manage complications that can include diabetes, high blood lipids, and accumulation of fat in the liver.”