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Exjade® (deferasirox; ICL670) NDA 21-882
Core Introduction (CI) 4/26/2017 4:34 PM DRA Introduction ppt Exjade® (deferasirox; ICL670) NDA United States Food and Drug Administration Blood Products Advisory Committee Meeting September 29, 2005
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Exjade® (deferasirox; ICL670) NDA 21-882 Introduction
Core Introduction (CI) 4/26/2017 4:34 PM DRA Introduction ppt Exjade® (deferasirox; ICL670) NDA Introduction PK Narang, PhD, FCP Vice President, Global Head Drug Regulatory Affairs—Oncology Novartis Pharmaceuticals Corporation
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Core Introduction (CI)
DRA Introduction ppt 4/26/2017 4:34 PM ICL670 Sponsor’s Agenda Introduction PK Narang, PhD, FCP Vice President, Drug Regulatory Affairs—Oncology Novartis Pharmaceuticals Corporation Iron Overload: Complications and Need for Therapy John B. Porter, MD Professor of Haematology University College, London, UK Efficacy and Safety Peter Marks, MD, PhD Senior Director, Oncology Clinical Development Novartis Pharmaceuticals Corporation Benefit/Risk Elliott Vichinsky, MD Director, Hematology/Oncology Children’s Hospital and Research Center at Oakland Oakland, California
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Iron Overload Overview
Core Introduction (CI) 4/26/2017 4:34 PM Iron Overload Overview DRA Introduction ppt Transfusional iron overload Serious complication in patients with transfusion- dependent anemias Significant morbidity/mortality for inadequately chelated patients Current “gold standard” therapy—Desferal® (Novartis) Only approved/available iron chelator in US Safe and effective (but parenteral) Poor compliance a significant issue Unmet medical need Safe, effective agent with positive benefit/risk
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Core Introduction (CI)
4/26/2017 4:34 PM ICL670 Overview A new class of tridentate iron chelator Orally dosed Drug kinetics support once-a-day dosing Efficacy similar to Desferal® Acceptable safety in adults and children
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ICL670 Proposed Indication
Core Introduction (CI) 4/26/2017 4:34 PM ICL670 Proposed Indication DRA Introduction ppt ICL670 is indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in adult and pediatric patients (≥ 2 years old)
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ICL670 Regulatory History
Core Introduction (CI) 4/26/2017 4:34 PM ICL670 Regulatory History IND submitted Orphan designation Fast-track status SPA for 0107, 0108, NDA accepted in CMA-1 program Jan 2005 NDA submission completed May 2005 Priority review granted Jun 2005 DRA Introduction ppt
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ICL670 Development Program Basis
Core Introduction (CI) 4/26/2017 4:34 PM ICL670 Development Program Basis DRA Introduction ppt β-thalassemia as a model for documenting “effectiveness” Largest prospective trials for an iron chelator Efficacy may be applicable to other conditions of transfusional iron overload including sickle cell disease Document safety in sickle cell disease and other rare anemias Provide sufficient pediatric data in pivotal trials 45% of patients
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Core Introduction (CI)
4/26/2017 4:34 PM Key ICL670 Efficacy and Safety Trials β-thalassemia and Other Rare Anemias DRA Introduction ppt Trial/Design N Treatments (duration) Patient population 0107 Phase 3 Randomized, open label, comparator-controlled 586 ICL670 5, 10, 20, 30 mg/kg vs DFO mg/kg (1 year) β-thalassemia Pediatric: (≥ 2 years old) and adult 0108 Phase 2 Open label, noncomparative 184 ICL670 5, 10, 20, 30 mg/kg (1 year) β-thalassemia/rare anemias 0109 195 Sickle cell disease Safety: Included study 106 (40 pediatric thalassemia patients). DFO = Desferal® (deferoxamine).
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ICL670 Questions & Answers
Core Introduction (CI) DRA Introduction ppt 4/26/2017 4:34 PM ICL670 Questions & Answers Consultants John Porter, MD Professor of Haematology University College, London Elliott Vichinsky, MD Director, Hematology/Oncology Children’s Hospital and Research Center at Oakland Alan Cohen, MD Physician-in-Chief and Medical Director, Thalassemia Program Children’s Hospital of Philadelphia Richard Larson, MD Professor of Medicine University of Chicago Raimund Hirschberg, MD Professor of Medicine Harbor-UCLA Medical Center Lloyd Fisher, PhD Professor Emeritus Department of Biostatistics University of Washington
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