Japanese filings for Shionogi's metreleptin and Otsuka's tolvaptin

Japanese drugmaker Shionogi (TYO: 4507) said last week that it has filed a New Drug Application with the Japanese Ministry of Health, Labor and Welfare for recombinant human leptin (generic name: metreleptin) for lipodystrophy. Metreleptin was in-licensed by Shionogi from USA-based Amylin Pharmaceuticals (Nasdaq: AMLN), which is currently the subject of a takeover by Bristol-Myers Squibb.

Lipodystrophy is a rare and life-threatening disorder characterized by a lack of required fat tissue for normal metabolic function, and is highly correlated to metabolic abnormalities such as severe diabetes, hypertriglyceridemia and fatty liver disease. There are currently no effective therapies that treat the underlying cause of the disease.
Leptin is a hormone secreted by fat tissue, which triggers the hypothalamus to suppress appetite. It is also known that leptin improves insulin resistance, glucose metabolism and lipid metabolism.

The Department of Medicine and Clinical Science at the Kyoto University Graduate School of Medicine, a research center of the Special Research Initiation for Drug Discovery for Intractable Diseases, and with assistance from the Translational Research Center at Kyoto University Hospital, conducted an investigator-initiated trial using metreleptin as a potential leptin-replacement therapy for lipodystrophy patients. The trial has concluded and, in July 2012, Shionogi filed an NDA for metreleptin for the treatment of lipodystrophy based on the results of the trial.

Otsuka files Samsca for additional indication