Metreleptin BLA Granted Priority Review for Rare Metabolic Disorders
Bristol-Myers Squibb and AstraZeneca announced that the FDA has accepted and granted Priority Review for the Biologics License Application (BLA) for metreleptin, an investigational recombinant analog of the human hormone leptin for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy (LD).
The primary data supporting the BLA filing are from the National Institutes of Health (NIH) ongoing, open-label trial in adult and pediatric patients with inherited or acquired LD. The trial was initiated in 2000 by investigators at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of NIH, to evaluate the safety and efficacy of investigational metreleptin for treating metabolic abnormalities associated with lipodystrophy, including insulin resistance, diabetes mellitus, hypertriglyceridemia and hepatic steatosis and steatohepatitis.
Metreleptin has previously received orphan designation from the FDA.