FDA Clears Orphan Drug Myalept for Rare Metabolic Disease

The U.S. Food and Drug Administration has cleared San Diego-based Amylin Pharmaceuticals' drug 'Myalept' to cure a rare metabolic disease.

The federal agency approved a new therapy Myalept (metreleptin for injection) as a replacement therapy to treat complications associated with deficiency of leptin for those battling with congenital or acquired generalized lipodystrophy.

Generalized lipodystrophy occurs when a person suffers with a loss of fat tissue and the patients with congenital generalized lipodystrophy are born with little or no fat tissues. People who acquire generalized lipodystrophy mostly lose the fat tissue over time. Since the hormone leptin is made of fat tissue, people with generalized lipodystrophy suffer with low levels of leptin.

Leptin plays a crucial role in controlling the intake of food as well regulates other hormones like insulin.

Those suffering with both the types of generalized lipodystrophy generally develop severe insulin resistance at a very early age leading to diabetes mellitus that is difficult to manage, or extremely high levels of trigylcerides in the blood (hypertriglyceridemia) that further trigger the inflammation of the pancreas.

"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," Mary Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA's Center for Drug Evaluation and Research, said in a statement.

In order to test the safety and effectiveness of the therapy, a study was conducted on 48 patients having either congenital or acquired generalized lipodystrophy. These patients also suffered with diabetes, hypertriglyceridemia or high levels of fasting insulin. At the end of the trial the researchers noticed a drop in HbA1c ( a measure of blood sugar control). They also noticed a reduction in the number of triglycerides and fasting glucose.

 The federal agency cautions the experts to evaluate the benefits of the treatment using Myalept in those with hematologic abnormalities or acquired generalized lipodystrophy.

The therapy is not approved for patients with HIV related lipodystrophy or those with other metabolic disease that include diabetes mellitus and hypertriglyceridemia.

The newly approved therapy is available only through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program, due to the risk of it developing anti drug antibodies and lymphoma. According to the REMS program, the person must be certified with the program by enrolling in and completing the training.

The agency demanded a post marketing tests for Myalept that includes a long term prospective observational study of patients treated using Myalept.