Reuters Health Information (2014-02-25): FDA approves drug for generalized lipodystrophy
FDA approves drug for generalized lipodystrophy
Last Updated: 2014-02-25 12:50:02 -0500 (Reuters Health)
(Reuters) - The US Food and Drug Administration has approved Myalept (metreleptin for injection) from Bristol-Myers Squibb to treat the complications of leptin deficiency in patients with generalized lipodystrophy, a condition associated with lack of fat tissue.
Patients with congenital generalized lipodystrophy are born with little or no fat tissue. Patients with acquired generalized lipodystrophy generally lose fat tissue over time.
Because the hormone leptin is made by fat tissue, patients with generalized lipodystrophy have very low leptin levels. Leptin regulates food intake and other hormones, such as insulin.
Patients with both types of generalized lipodystrophy often develop severe insulin resistance at a young age and may have diabetes mellitus that is difficult to control or 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," Dr. Mary Parks of the FDA said in a statement.
The safety and effectiveness of Myalept, an analog of leptin made through recombinant DNA technology, were evaluated in an open-label, single-arm study that included 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes mellitus, hypertriglyceridemia, and/or elevated levels of fasting insulin. The trial showed reductions in HbA1c (a measure of blood sugar control), fasting glucose, and triglycerides.
Anti-drug antibodies with neutralizing activity to leptin and/or Myalept may develop, which could result in severe infections or loss of treatment effectiveness, the FDA notes.
Because of the risks associated with the development of neutralizing antibodies and lymphoma, Myalept is available only through the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program.