The US Food and Drug Administration’s (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) has recommended AstraZeneca and Bristol-Myers Squibb‘s (BMS) investigational medicine metreleptin for the treatment of paediatric and adult patients with generalised lipodystrophy (LD).
EMDAC has been determined in particular by a vote of 11 to one, under there being substantial evidence that the benefits of metreleptin exceed the risks for treatment.
LD is a group of rare syndromes related with severe metabolic abnormalities and significant morbidity and mortality.
Metreleptin is not recommended by EMDAC in patients with partial LD for the indication currently proposed, by a vote of two to ten.
Both companies remain committed to pursuing metreleptin for treatment in patients with metabolic disorders associated with partial LD. They 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 licence application (BLA) for metreleptin.
The FDA is currently reviewing metreleptin as a treatment of paediatric and adult patients with generalised lipodystrophy (LD) or metabolic disorders associated with partial LD, including hypertriglyceridemia and/or diabetes mellitus inadequately controlled on a current therapy, and evidence of hepatic steatosis (fatty liver disease).
So far there, are no therapies approved by the FDA for treatment of patients with rare forms of LD (not including HIV-associated LD) and the Prescription Drug User Fee Act (PDUFA) goal date for metreleptin is 24 February 2014.
Recommendations from EMDAC were based on a review of data from the metreleptin clinical development programme for LD that supported the BLA submission. This included pivotal efficacy and safety data from two open-label, investigator-sponsored National Institutes of Health (NIH) studies.
The approval is also based on an important supplemental efficacy and safety data on investigational metreleptin from an additional open-label expanded access trial, FHA101.
Metreleptin has also received orphan designation from the FDA and the European Medicines Agency (EMA).