FDA approves Myalept for Lipodystrophy-AstraZeneca

The FDA has approved Myalept (metreleptin for injection), from AstraZeneca, which is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized Lipodystrophy. Myalept, a recombinant analog [laboratory-created form] of human leptin, is the first and only treatment approved by the FDA for these patients. The safety and effectiveness of Myalept for the treatment of complications of partial Lipodystrophy or for the treatment of liver disease, including non-alcoholic steatohepatitis (NASH), have not been established.

The drug is not indicated for use in patients with HIV-related lipodystrophy or for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of congenital or acquired generalized lipodystrophy. In an open label, single arm study (N=48), the most common adverse reactions reported in ≥5% in patients with generalized lipodystrophy receiving Myalept were: Headache (13%), hypoglycemia (13%), decreased weight (13%), abdominal pain (10%) and arthralgia (8%).