Metreleptin sub analysis in paediatric patients with Lipodystrophy

BMS and AstraZeneca announce the results from a 12-month sub-group analysis of a National Institutes of Health (NIH), open-label, long-term research study of metreleptin, an investigational agent for the treatment of metabolic disorders associated with inherited or acquired Lipodystrophy (LD), a rare disease estimated to affect a few thousand people around the world, often with an early age of onset.

In this analysis, which involved 39 pediatric LD patients less than 18 years of age at the time of study enrollment, investigational metreleptin treatment led to mean reductions in HbA1c (average blood sugar levels over three months) and triglyceride levels, as well as mean reductions in liver function tests (including alanine aminotransferase, or ALT, and aspartate aminotransferase, or AST). The full study was initiated in 2000 by investigators at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the NIH, and is currently ongoing. Results from the pediatric analysis were presented at the 2013 Pediatric Endocrine Society (PES) Annual Meeting, being held in conjunction with the 2013 Pediatric Academic Societies (PAS) Annual Meeting in Washington, D.C.