FDA's Orphan Drug Act was designed to encourage the development of products that demonstrate promise for the diagnosis, prevention and/or treatment of life-threatening or very serious conditions that are rare and affect 200,000 persons or less in the United States. Orphan drug designation provides an important economic incentive for the development of new products in the cancer field. U.S. orphan drug designation provides for seven years of market exclusivity, reduction in regulatory fees, and additional regulatory support for R&D initiatives.
“The decision by the FDA to grant Amrubicin orphan drug designation in this critical area of oncology advances our efforts to deliver innovative therapies to patients in areas of unmet medical need and represents the first such milestone in the area of solid tumor cancers,” said Graham Burton M.D., SVP, Global Regulatory Affairs and Pharmacovigilance for Celgene Corporation. “We will continue to work diligently with regulators as we advance this promising next generation anthracycline through clinical development.”
About Amrubicin
Amrubicin is a third-generation, synthetic anthracycline analogue that has demonstrated substantial clinical efficacy in the treatment of small cell lung cancer. Amrubicin is a potent topoisomerase II inhibitor and is being studied as a single agent and in combination with anti-cancer therapies for a variety of solid tumors, including lung and breast cancers.
Amrubicin is currently approved and marketed in Japan for the treatment of small cell lung cancer by Nippon Kayaku, a Japanese pharmaceutical firm focused on oncology, which licensed Japanese marketing rights from Dainippon Sumitomo, the original developer of the therapy. Dainippon Sumitomo also licensed the U.S. and European rights of Amrubicin to Pharmion Corporation, which was acquired by Celgene Corporation in 2008.
About Small Cell Lung Cancer
Small cell lung cancer is a disease in which malignant cells form in the tissues of the lung, and which occurs almost exclusively in people who smoke. While small cell lung cancer constitutes approximately 15 percent of all lung cancers, SCLC tends to be more aggressive and fast growing than the more common non-small cell lung cancer. Of the estimated 65,000 patients diagnosed with SCLC each year in the US and EU, approximately 60 percent of patients have extensive disease at diagnosis, and the remaining 40 percent present with localized, or limited stage, disease.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at www.celgene.com.
This release contains forward-looking statements which are subject to known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations expressed or implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and other factors described in the Company's filings with the Securities and Exchange Commission such as our 10K, 10Q and 8K reports.
Contact: Celgene Corporation David Gryska, 908-673-9059 Senior Vice President and Chief Financial Officer or Brian P. Gill, 908-673-9530 Vice President Corporate Communications
Source: Celgene Corporation
