FDA Advisory Committee Recommends Metreleptin for Generalised Lipodystrophy
An FDA advisory committee has recommended the investigational medicine metreleptin for treating generalised lipodystrophy in paediatric and adult patients. The committee did not recommend it for partial lipodystrophy in its current proposed indication.
The US Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) has recommended metreleptin, an investigational medicine, for the treatment of generalised lipodystrophy (LD) in paediatric and adult patients. The committee voted 11 to 1 that there is substantial evidence that the benefits of metreleptin outweigh the risks for this patient group.
However, the EMDAC did not recommend metreleptin for patients with partial lipodystrophy under the currently proposed indication, with a vote of 2 to 10. AstraZeneca and Bristol-Myers Squibb Company stated they remain committed to working with the FDA to identify appropriate patients with partial LD who may benefit from the drug.
Lipodystrophy is a group of rare syndromes often associated with severe metabolic abnormalities, significant morbidity, and mortality. There are currently no FDA-approved therapies for rare forms of LD, excluding HIV-associated LD. Metreleptin is being reviewed for treating generalised LD or metabolic disorders associated with partial LD, such as hypertriglyceridemia and inadequately controlled diabetes.
The FDA is not bound by the advisory committee's recommendation but will consider it during its review of the Biologics License Application (BLA) for metreleptin. The Prescription Drug User Fee Act (PDUFA) goal date for the FDA's decision is February 24, 2014.