The FDA has approved the use of Myalept (metreleptin for injection) as replacement therapy to treat the complications of a lack of fat tissue known as congenital generalized lipodystrophy (meaning patients re born with the condition) or acquired generalized lipodystrophy (in which they lose it over time). Because of this, their bodies end up with a deficiency in the hormone leptin, which regulates food intake and other hormones, including insulin. This in turn causes patients with both types of the disease to develop severe insulin resistance when young making it difficult for them to control or very high levels of triglycerides in the blood (hypertriglyceridemia) that can lead to 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,” said Mary Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research.
Dr. Parks also noted that “anti-drug antibodies with neutralizing activity to leptin and/or Myalept may develop, which could result in severe infections or loss of treatment effectiveness,” as well as the fact that cases of T-cell lymphoma have been reported in patients with acquired generalized lipodystrophy (both treated and not treated with Myalept).
Because of this, Myalept is only available through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program, which requires that all prescribers must be certified by enrolling in and completing training
It should also be noted that Myalept is not approved for use in patients with HIV-related lipodystrophy or in not approved for use in patients with HIV-related lipodystrophy or in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.
For more information readers can contact the FDA at 888-INFO-FDA.