AstraZeneca (NYSE:AZN) and Bristol-Myers Squibb Company (NYSE:BMY) today announced the U.S. Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) recommends the investigational medicine metreleptin for the treatment of pediatric and adult patients with generalized lipodystrophy (LD). Specifically, EMDAC determined by a vote of 11 to 1 that there is substantial evidence that the benefits of metreleptin exceed the risks for the treatment of pediatric and adult patients with generalized lipodystrophy.
EMDAC did not recommend metreleptin in patients with partial LD for the indication currently proposed, by a vote of 2 to 10. AstraZeneca and Bristol-Myers Squibb remain committed to pursuing metreleptin for treatment in patients with metabolic disorders associated with partial LD. The Companies acknowledge the committee's feedback and will continue to work with the FDA to identify the appropriate patients with partial LD who may benefit from metreleptin.
The FDA is not bound by the EMDAC's recommendation but will take it into consideration when reviewing the Biologics License Application (BLA) for metreleptin.
LD is a group of rare syndromes often associated with severe metabolic abnormalities and significant morbidity and mortality. Metreleptin is being reviewed by the FDA as a treatment of pediatric and adult patients with generalized lipodystrophy (LD) or metabolic disorders associated with partial LD, including hypertriglyceridemia and/or diabetes mellitus inadequately controlled on a current therapy, and/or evidence of hepatic steatosis.
The Prescription Drug User Fee Act (PDUFA) goal date for metreleptin is February 24, 2014. There are no therapies approved by the FDA to treat patients with rare forms of LD (not including HIV-associated LD).
The EMDAC based its recommendations on a review of data from the metreleptin clinical development program for LD that supported the BLA submission, including pivotal efficacy and safety data from two open-label, investigator-sponsored National Institutes of Health (NIH) studies, as well as important supplemental efficacy and safety data on investigational metreleptin from an additional open-label expanded access study, FHA101.