Advisors to the US Food and Drug Administration have backed use of AstraZeneca and Bristol-Myers Squibb's lipodystrophy drug metreleptin for one indication but not for another.
The agency's Endocrinologic and Metabolic Drugs Advisory Committee voted 11-1 in favor of the use of metreleptin in the treatment of paediatric and adult patients with generalised LD. However, the panel voted 10-2 against recommending the therapy for patients with metabolic disorders associated with partial LD.
LD is a group of rare syndromes which affect only a few thousand people worldwide, which results in a loss of fat tissue that causes a deficit in the hormone leptin.
AstraZeneca and B-MS said they 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 Prescription Drug User Fee Act goal date for the drug, which B-MS got access to through its acquisition of Amylin and then partnered with AstraZeneca, is February 24.