U.S. FDA Grants Priority Review to Bristol-Myers Squibb and AstraZeneca's Metreleptin, an Investigational Agent for Treatment of Metabolic Disorders Associated with Rare Forms of Lipodystrophy
Tuesday, June 4, 2013 9:03 am EDT
"The FDA's granting of Priority Review for the metreleptin BLA highlights the significant unmet medical need for patients affected by lipodystrophy and brings us an important step closer to potentially bringing a treatment option to these patients,"
PRINCETON, N.J. & WILMINGTON, Del.--(BUSINESS WIRE)--Bristol-Myers Squibb Company (NYSE: BMY) and AstraZeneca (NYSE: AZN) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted a Priority Review designation for the Biologics License Application (BLA) for 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.
"The FDA's granting of Priority Review for the metreleptin BLA highlights the significant unmet medical need for patients affected by lipodystrophy and brings us an important step closer to potentially bringing a treatment option to these patients," said Fred Fiedorek, MD, senior vice president, Head of Development, CV and Metabolics at Bristol-Myers Squibb. "Developing metreleptin for the treatment of lipodystrophy demonstrates Bristol-Myers Squibb and AstraZeneca's continued dedication to scientific innovation and global patient care for people impacted by diabetes and related metabolic disorders."
The FDA grants Priority Review to medicines that may provide a treatment option where little or no adequate therapy exists. Under the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete its review within an eight-month review cycle, rather than the standard 12-month review cycle.
The primary data supporting the BLA filing are from the National Institutes of Health (NIH) ongoing, open-label trial in adult and pediatric patients with inherited or acquired LD.
About the Ongoing National Institutes of Health (NIH) Study
The NIH study is an ongoing, open-label trial of investigational metreleptin in patients with inherited or acquired lipodystrophy. The trial was initiated in 2000 by investigators at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of NIH, to evaluate the safety and efficacy of investigational metreleptin for treating metabolic abnormalities associated with lipodystrophy, including insulin resistance, diabetes mellitus, hypertriglyceridemia and hepatic steatosis and steatohepatitis (also known as fatty liver disease).
Metreleptin, an investigational recombinant analog of the human hormone leptin, has received orphan designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and is being evaluated for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy.
Lipodystrophy is a very rare disease estimated to affect only a few thousand people around the world. The condition often presents at an early age during childhood or adolescence.
Patients with lipodystrophy experience a loss of fat tissue, especially fat under the skin. This loss of fat tissue causes a deficit in the hormone leptin. Without enough fat tissue or leptin, the body's system for regulating energy use and storage falls out of balance. The resulting serious imbalance causes lipid to accumulate where it shouldn't be found—such as in the liver and muscle—which can lead to serious complications, including severe insulin resistance, diabetes, high levels of triglycerides and/or inflammation in the liver.
There are several reasons for developing lipodystrophy. In some patients, it is genetic and in others it may be acquired for different reasons, including cases in which the immune system may attack and destroy existing fat tissue. Sometimes, clearly defined reasons for the development of the condition are unknown.
Bristol-Myers Squibb and AstraZeneca Collaboration
Bristol-Myers Squibb and AstraZeneca entered into a collaboration in January 2007 to research, develop and commercialize select investigational drugs for type 2 diabetes. The Bristol-Myers Squibb/AstraZeneca collaboration is dedicated to global patient care, improving patient outcomes and creating a new vision for the treatment of diabetes and related metabolic disorders. The expansion of the collaboration covers the co-development and marketing of products in the Amylin Pharmaceuticals portfolio, including, among others, investigational metreleptin, a recombinant analog of the human hormone leptin currently under review by the U.S. Food and Drug Administration (FDA) for the treatment of lipodystrophy.
About Bristol-Myers Squibb
Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information, please visit http://www.bms.com or follow us on Twitter at http://twitter.com/bmsnews.
AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.com.
Bristol-Myers Squibb Forward Looking Statement
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding product development. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among other risks, there can be no guarantee that metreleptin will receive FDA approval, that the timing of any such approval will occur within the time period described in this release or that, if approved, it will become a commercially successful product. Forward-looking statements in this press release should be evaluated together with the many uncertainties that affect Bristol-Myers Squibb's business, particularly those identified in the cautionary factors discussion in Bristol-Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2012, in our Quarterly Reports on Form 10-Q and our Current Reports on Form 8-K. Bristol-Myers Squibb undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.
AstraZeneca Cautionary Statement Regarding Forward-Looking Statements
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