FDAnews Drug Daily Bulletin

FDA Advisors Back Approval of Investigational Lipodystrophy Drug

Dec. 13, 2013

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee on Dec. 11 voted 11-1 to recommend approval of AstraZeneca and Bristol-Myers Squibb’s investigational recombinant analog metreleptin for adults and children with generalized lipodystrophy.

Lipodystrophy constitutes a group of rare syndromes often associated with severe metabolic abnormalities and significant morbidity and mortality, AstraZeneca says. There are no therapies approved in the U.S. to treat rare forms of lipodystrophy (not including HIV-associated LD).

The advisory panel’s decision was based on safety and efficacy data from two ongoing open-label, investigator-sponsored studies and supplemental data from a completed open-label expanded-access study.

The FDA is reviewing the companies’ BLA for metreleptin for adults and pediatric indications for generalized lipodystrophy or metabolic disorders associated with partial lipodystrophy, such as hypertriglyceridemia or diabetes mellitus uncontrolled on current therapies and/or fatty liver disease. Agency advisors voted 2-10 against recommending the drug for partial lipodystrophy.

Metreleptin’s PDUFA date is Feb. 24. — Lena Freund

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