Metreleptin was approved in the United States as adjunct to diet as replacement therapy to
treat the complications of leptin deficiency in patients with congenital or acquired
generalized lipodystrophy in February 2014. The approval was based on results obtained in 2
open-label, investigator-sponsored studies (Studies 991265 and 20010769) conducted at the
National Institutes of Health (NIH) to evaluate the safety and efficacy of metreleptin
treatment in patients with lipodystrophy and 1 treatment IND (FHA101/MB002-002/MB002-002)
conducted by Bristol-Myers Squibb on behalf of AstraZeneca (BMS/AZ) in patients with diabetes
mellitus and/or hypertriglyceridemia related to lipodystrophy. These studies enrolled
patients with lipodsytrophy including both generalized and partial lipodystrophy. Although
the marketing authorization restricted the indication to patients with generalized
lipodystrophy, meaningful clinical benefit was achieved in a subset of patients with partial
lipodystrophy, and these patients from FHA101/MB002-002 form the basis of the request for
ongoing treatment under expanded access.