Metreleptin Shows Promise for Pediatric Lipodystrophy

Bristol-Myers Squibb and AstraZeneca announced 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 human hormone leptin analogue for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy (LD).

This analysis included 39 pediatric LD patients <18 years of age at the time of enrollment. It was conducted to examine the effects of metreleptin on metabolic parameters associated with LD, including HbA1c, triglyceride levels and liver function tests, in pediatric patients enrolled in the NIH study. The four major subtypes of LD, congenital generalized LD, acquired generalized LD, familial partial LD and acquired partial LD were studied. Patients were assigned to one of two groups: children (≤12 years) or adolescents (>12 to <18). All data was analyzed at month 12 of metreleptin treatment, if available (n=27).

In adolescents with LD, metreleptin treatment resulted in statistically significant reductions in elevated HbA1c (mean HbA1c decreased from 9.8±1.8% at baseline to 7.7±1.7% at month 12; mean decrease of 2.3±0.4%) and elevated triglycerides (mean triglyceride concentrations decreased from 1378±2024 mg/dL at baseline to 385±446 mg/dL at month 12; mean decrease of 44±14%). Elevated liver function tests also decreased (mean ALT decreased from 105±97 U/L at baseline to 59±108 U/L at month 12; mean decrease of 46±40%, and mean AST decreased from 87±89 U/L at baseline to 57±118 U/L at month 12; mean decrease of 31±38%). In younger children with LD, mean liver function tests were markedly elevated at baseline and decreased with investigational metreleptin treatment (mean ALT decreased from 193±202 U/L at baseline to 155±274 U/L at month 12; mean decrease of 38±47%, and AST decreased from 119±112 U/L at baseline to 90±144 U/L at month 12; mean decrease of 30±29%). Reductions in ALT and AST were clinically meaningful even though they did not reach statistical significance. The study by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a part of the NIH, is currently ongoing.

Metreleptin has previously received Orphan Drug designation from the FDA.

For more information visit or