FDA clears Bristol drug for rare fat disorder
The Food and Drug Administration has approved the first drug to treat a rare metabolic disorder characterized by abnormally low fat levels.
The agency on Tuesday cleared Bristol-Myers Squibb's Myalept for patients with lipodystrophy, a condition in which people are born with little or no fat tissue. The biotech drug mimics the effect of the natural hormone leptin, which helps regulate metabolism, appetite and insulin production.
Patients with lipodystrophy often develop diabetes at a young age and have difficulty controlling levels of fats known as triglycerides, which can lead to inflammation of the pancreas.
Serious side effects have been connected with Myalept use, including drug resistance and lymphoma. As a result, the drug will only be available from a limited number of health care professionals who are certified in prescribing the drug.
Myalept was developed by San Diego-based Amylin Pharmaceuticals, which was acquired by Bristol-Myers Squibb Company in 2012.