Amylin Submits Clinical and Nonclinical Sections of Rolling Biologics License Application for Metreleptin to Treat Rare Forms of Lipodystrophy
SAN DIEGO, Dec. 20, 2010 /PRNewswire-FirstCall/ -- Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) today announced that it has submitted the initial sections of a rolling submission for a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the use of metreleptin to treat diabetes and/or hypertriglyceridemia (high levels of triglycerides in the bloodstream) in patients with rare forms of lipodystrophy. Consistent with the severity and rare nature of the disorder, Amylin has received both orphan drug designation from FDA's Office of Orphan Products Development, as well as Fast Track designation for the use of metreleptin in patients with lipodystrophy. The focus of this marketing application is on rare inherited and acquired forms of lipodystrophy.
In the first part of its rolling submission, the Company submitted the nonclinical and clinical sections of the BLA. The Company plans to submit the chemistry, manufacturing and controls (CMC) section of the BLA by the end of 2011, which will complete the submission.
"It is gratifying to see that, after years of research focused on leptin as an effective therapy for lipodystrophy, we are now closer to bringing this important and innovative medicine to patients who are in dire need of better treatments," said Phillip Gorden, M.D., Director Emeritus, Senior Investigator, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the National Institutes of Health (NIH). Dr. Gorden is the principal investigator of an ongoing NIH clinical study evaluating the long-term efficacy of metreleptin treatment in lipodystrophy.
Lipodystrophy syndromes are characterized by abnormalities in adipose (fat) tissue distribution with loss of subcutaneous fat, and often manifest in childhood or adolescence. Patients with lipodystrophy can have multiple severe metabolic abnormalities, includin