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 lipodystrophy 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.
Leptin is a naturally occurring hormone and an important regulator of energy homeostasis and other diverse physiological functions. Circulating levels of leptin closely correlate with the amount of adipose tissue present. Metreleptin, a recombinant analogue of human leptin, is a 147-amino acid polypeptide that differs from the human leptin sequence by 1 additional amino acid, methionine, located at the amino-terminal end. Metreleptin has the same physiological effects as leptin, including regulation of energy homeostasis and metabolic function. The patient group covered under this expanded access submission has demonstrated evidence of clinical benefit from treatment with metreleptin in clinical study FHA101/MB002-002, and needs expanded access to continue treatment without interruption. New enrollment, subject to approval by the FDA, can be considered on a case-by-case basis.
Study Type
EXPANDED_ACCESS
Metreleptin open-label treatment
University of Michigan
Ann Arbor, Michigan, United States
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