Table of Contents
Table of Contents 2
List of Tables 7
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Aplastic Anemia Overview 9
Therapeutics Development 10
An Overview of Pipeline Products for Aplastic Anemia 10
Aplastic Anemia Therapeutics under Development by Companies 12
Aplastic Anemia Therapeutics under Investigation by Universities/Institutes 13
Late Stage Products 15
Comparative Analysis 15
Mid Clinical Stage Products 16
Comparative Analysis 16
Early Clinical Stage Products 17
Comparative Analysis 17
Pre-Clinical Stage Products 18
Comparative Analysis 18
Aplastic Anemia Therapeutics – Products under Development by Companies 19
Aplastic Anemia Therapeutics – Products under Investigation by Universities/Institutes 20
Companies Involved in Aplastic Anemia Therapeutics Development 23
Aspen Pharmacare Holdings Ltd 23
Aplastic Anemia – Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Combination Products 25
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 31
Busulfan + Cyclophosphamide + Allogeneic Stem Cell Transplantation - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Cyclophosphamide + Total Body Radiation - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Fludarabine + Melphalan + Allogeneic Stem Cell Transplantation - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Cyclophosphamide + Etoposide + Total Body Irradiation - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Carboplatin + Thiotepa + Cyclophosphamide + Allogeneic Stem Cell Transplantation - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Anti-Thymocyte Globulin + Busulfan + Cyclophosphamide - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Anti-Thymocyte Globulin + Cyclophosphamide + Allogeneic Stem Cell Transplantation - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Anti-Thymocyte Globulin + Cyclophosphamide + Total Body Irradiation + Allogeneic Stem Cell Transplantation - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 41
Campath-1H + Cyclophosphamide + Fludarabine + Radiation Therapy + Stem Cell Transplantation - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Busulfan + Melphalan + Campath + Peripheral Blood Stem Cell Transplantation - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Anti-Thymocyte Globulin + Fludarabine + Cyclophosphamide + CD3±CD19 Depleted Hematopoietic Stem Cell Transplantation - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Cyclophosphamide + Leucovorin + Methotrexate + Allogeneic Bone Marrow Transplantation + Radiation Therapy - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 48
Anti-Thymocyte Globulin + Cyclophosphamide + Fludarabine + Peripheral Blood Stem Cell Transplantation - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Anti-Thymocyte globulin + G-CSF + Cyclophosphamide + Fludarabine + Umbilical Cord Blood Transplantation + Methylprednisolone - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 52
Alemtuzumab + Cyclosporine - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Rituxan - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Cyclophosphamide - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Antithymocyte Globulin + Cyclosporine + Mycophenolate Mofetil - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Anti Thymocyte Globulin + Cyclosporine + Rapamune - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Promacta - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Antithymocyte Globulin + Cyclophosphamide + Fludarabine + Radiation Therapy + Allogeneic Marrow Transplantation - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 63
Cyclophosphamide + Fludarabine Phosphate + Anti-thymocyte Globulin + Total Body Irradiation + Umbilical Cord Blood Stem Cells + Haploidentical Peripheral Blood CD34+ Cells - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 65
ATG + Gengraf - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Cyclophosphamide + Fludarabine + Radiation Therapy + Umbilical Cord Blood Stem Cell Transplantation - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Cyclophosphamide + Pentostatin + Radiation Therapy + Umbilical Cord Blood Stem Cell Transplantation - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Cyclophosphamide + Fludarabine Phosphate + Thymoglobulin - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Zenapax - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
Zenapax - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Cyclophosphamide + Fludarabine + Cyclosporine + Campath-1H + GM-CSF - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 75
Rabbit Antithymocyte Globulin - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
Campath 1H - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF - Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 79
Antithymocyte Globulin + Cyclosporine A + Mycophenolate Mofetil - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
Cyclophosphamide + Radiation Therapy + Allogeneic Stem Cell Transplantation - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
Anti-Thymocyte Globulin + Cyclophosphamide + Fludarabine + Methylprednisolone + Mycophenolate Mofetil + Prednisone + Tacrolimus + Umbilical Cord Blood Transplantation + Peripheral Blood Stem Cell Transplantation - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 86
Anti-Thymocyte Globulin + Graft-Versus-Tumor Induction Therapy + GM-CSF + Therapeutic Allogeneic Lymphocytes + Cyclophosphamide + Fludarabine + Methylprednisolone + Mycophenolate Mofetil + Tacrolimus + Allogeneic Peripheral Blood Stem Cell Transplantation + Peripheral Stem Cell Transplantation + Umbilical Cord Blood Transplantation - Drug Profile 87
Product Description 87
Mechanism of Action 88
R&D Progress 89
Campath-1H - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
Fludarabine + Cyclophosphamide - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
Cyclosporine + Thymoglobulin - Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 92
Bone Marrow Mesenchymal Stem Cells - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
Bone Marrow Derived Mesenchymal Stem Cells - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
AP032 - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
Aplastic Anemia Therapeutics – Drug Profile Updates 96
Aplastic Anemia Therapeutics – Discontinued Products 97
Aplastic Anemia Therapeutics - Dormant Products 98
Appendix 99
Methodology 99
Coverage 99
Secondary Research 99
Primary Research 99
Expert Panel Validation 99
Contact Us 100
Disclaimer 100
List of Tables
Number of Products Under Development for Aplastic Anemia, H1 2012 10
Products under Development for Aplastic Anemia – Comparative Analysis, H1 2012 11
Number of Products under Development by Companies, H1 2012 12
Number of Products under Investigation by Universities/Institutes, H1 2012 14
Comparative Analysis by Late Stage Development, H1 2012 15
Comparative Analysis by Mid Clinical Stage Development, H1 2012 16
Comparative Analysis by Early Clinical Stage Development, H1 2012 17
Comparative Analysis by Pre-Clinical Stage Development, H1 2012 18
Products under Development by Companies, H1 2012 19
Products under Investigation by Universities/Institutes, H1 2012 20
Products under Investigation by Universities/Institutes, H1 2012 (Contd..1) 21
Products under Investigation by Universities/Institutes, H1 2012 (Contd..2) 22
Aspen Pharmacare Holdings Ltd, H1 2012 23
Assessment by Monotherapy Products, H1 2012 24
Assessment by Combination Products, H1 2012 25
Assessment by Stage and Route of Administration, H1 2012 27
Assessment by Stage and Molecule Type, H1 2012 30
Aplastic Anemia Therapeutics – Drug Profile Updates 96
Aplastic Anemia Therapeutics – Discontinued Products 97
Aplastic Anemia Therapeutics – Dormant Products 98
List of Figures
Number of Products under Development for Aplastic Anemia, H1 2012 10
Products under Development for Aplastic Anemia – Comparative Analysis, H1 2012 11
Products under Development by Companies, H1 2012 12
Products under Investigation by Universities/Institutes, H1 2012 13
Late Stage Products, H1 2012 15
Mid Clinical Stage Products, H1 2012 16
Early Clinical Stage Products, H1 2012 17
Pre-Clinical Stage Products, H1 2012 18
Assessment by Monotherapy Products, H1 2012 24
Assessment by Combination Products, H1 2012 25
Assessment by Route of Administration, H1 2012 26
Assessment by Stage and Route of Administration, H1 2012 27
Assessment by Molecule Type, H1 2012 28
Assessment by Stage and Molecule Type, H1 2012 29