On May 02, 2016 Kite Pharma, Inc. (Nasdaq:KITE) ("Kite"), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) granted orphan drug designations to Kite’s lead product candidate, KTE-C19, for the treatment of primary mediastinal B cell lymphoma (PMBCL), mantle cell lymphoma (MCL), follicular lymphoma (FL), acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL) (Press release, Kite Pharma, MAY 2, 2016, View Source [SID:1234511757]). Kite previously received orphan drug designation for KTE-C19 for the treatment of diffuse large B cell lymphoma (DLBCL) in both the U.S. and the EU, as well as orphan drug designations in the EU for PMBCL, MCL, FL, ALL, and CLL. Schedule your 30 min Free 1stOncology Demo! "Kite has now secured Orphan Drug Designations in both the U.S. and the EU for the leading indications in hematological malignancies. This is an important regulatory milestone as we further our development of KTE-C19 in advanced hematological cancers, including the planned initiation of clinical studies in CLL and FL patients in 2017," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We are encouraged by the progress of our four ongoing ZUMA clinical trials and our ability to manufacture clinical supply of KTE-C19 in our own Santa Monica facilities. We look forward to working closely with the FDA to bringing this potentially transformative therapy to patients with a significant unmet need."
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Orphan drug designation is granted by the FDA’s Office of Orphan Products Development (OOPD) to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The OOPD provides incentives for sponsors to develop products for rare diseases which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same biologic for the same indication for seven years, except in limited circumstances.