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Global Markets for Orphan Drugs – Focus on Biologics

  • June 2013
  • -
  • BCC Research
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  • 150 pages

INTRODUCTION

STUDY OBJECTIVES

The orphan drug market is one of the fastest-growing segments in the pharmaceutical and biotechnology industry. BCC’s goal in conducting this study is to provide an overview of the current and future global market for orphan drugs. The key objective is to present a comprehensive analysis of current orphan drugs and those under development as important therapies and treatments for rare diseases.

This report explores present and future strategies and regulations within the orphan drug market, advances in the market, the setbacks, the current situation and the needs of the market. The market for each drug listed by its generic name/active ingredient and approved indication(s) is also analyzed in this report.

A detailed analysis of the structure of the orphan drug industry has been conducted. Revenues are broken down by region drug type (i.e., biological and nonbiological), active ingredient and area of medical application. Sales figures are estimated for the five-year period from 2012 through 2017.

Applications for orphan drugs are also discussed in the report, with an emphasis on the usage of the drugs in various disease segments. The report also covers significant patents and their allotments in each category.

REASONS FOR DOING THIS STUDY

Extensive research in the field of orphan drugs has resulted in therapeutic treatments for a number of rare diseases. Most of the big pharmaceutical companies have now begun to explore the advantages of orphan drugs as a major source of profit and a steady market.

R&D spending, competition and new technologies are increasing. Advances, new product launches and assistance from regulatory bodies are positively impacting the growth of the market for orphan drugs in the near term. This study examines these factors.

This study focuses on the manufacturers and users of orphan drugs and its market growth. Pharmaceutical biotechnical companies, research institutes and physicians will find this study to be of interest.

SCOPE OF THE STUDY

The scope of this study includes orphan drugs in pharmaceutical markets worldwide. BCC analyzes each market and its applications, regulatory environment, technology, market projections and market shares. Technological issues include the latest trends and developments. The emerging market for orphan drugs includes countries such as India, China, Japan, Korea, Taiwan, Canada, Africa, Australia and New Zealand.

METHODOLOGY

Both primary and secondary research methodologies were used in preparing this study. BCC conducted a comprehensive literature search, which included technical newsletters and journals and many other sources. Projections were based on estimates such as the current number of end users, potential end users, mergers and acquisitions and market trends.

INFORMATION SOURCES

Many companies were surveyed to obtain data for this study. Included were manufacturers and end users of orphan drugs within the therapeutic categories examined and various disease sector industries. Data were gathered from various industry sources. BCC spoke with officials within the industry, consulted newsletters, company literature, product literature and a host of technical articles, journals, indexes and abstracts. Exhaustive investigations of databases by key terminology were completed. In addition, data were compiled from current financial, trade and government sources.

ABOUT THE AUTHOR

A team of professionals in the biotechnology industry carried out this study. The project manager is Shalini S. Dewan, who holds a master’s degree in pharmaceutical chemistry and has more than 14 years of industry experience. Among the research topics she has covered are studies on compounds of potential pharmaceutical interest from ibuprofen and 2-naphthyl acetic acid. Shalini was awarded a Gold Medal by the Prime Minister of India for her work and has worked with top companies in India and in the U.S. Some of the other reports she has prepared for BCC include Reagents for Chromatography; Spectroscopy: An Enduring Market; Advanced Drug Delivery Systems: Technologies and Global Markets; Orthopedic Drugs, Implants and Devices; Medical Imaging Reagents and Analysis Equipment; Pharmaceutical Regulatory Industry; The Dynamic Media, Sera and Reagent Market in Biotechnology; Contract Pharmaceutical Manufacturing, Research and Packaging: Global Markets; Chiral Technology: Global Markets; Autacoids and Related Drugs: Technologies and Global Markets; Contraceptives: Technologies and Global Markets; Liver Disease Treatments: The Global Market; Hormone Replacement Therapies and Other Hormone Therapies: Global Markets; Cardiovascular Medicine: Diagnostics, Drugs and Devices; and Cancer Therapies: Technologies and Global Markets.

Table Of Contents

Global Markets for Orphan Drugs - Focus on Biologics
TABLE OF CONTENTS

CHAPTER 1 INTRODUCTION 2
STUDY OBJECTIVES 2
REASONS FOR DOING THIS STUDY 2
CONTRIBUTIONS OF THE STUDY AND INTENDED AUDIENCE 2
SCOPE OF THE STUDY 3
METHODOLOGY 3
INFORMATION SOURCES 3
ABOUT THE AUTHOR 3
RELATED BCC RESEARCH REPORTS 4
BCC ONLINE SERVICES 4
DISCLAIMER 4
CHAPTER 2 EXECUTIVE SUMMARY 6
SUMMARY TABLE GLOBAL SALES OF ORPHAN DRUGS BY TYPE, THROUGH 2017 ($
MILLIONS) 8
SUMMARY FIGURE GLOBAL SALES OF ORPHAN DRUGS BY TYPE, 2010-2017 ($
MILLIONS) 8
CHAPTER 3 OVERVIEW 10
TABLE 1 HISTORY OF ORPHAN DRUG LEGISLATION 11
U.S. ORPHAN DRUG ACT 11
REASONS FOR THE ORPHAN DRUG ACT 11
OTHER LEGISLATION IN THE U.S. 12
ORPHAN DRUG PROGRAMS 12
Orphan Drug Designation Program 12
Orphan Products Grant Program 13
Pediatric Device Consortia (PDC) Grant Program 13
Humanitarian Use Device (HUD) Program 13
TABLE 2 U.S. ORPHAN DRUG ACT, AMENDMENTS AND OTHER LEGISLATION 13
SUCCESS OF ORPHAN DRUG ACT 14
TABLE 3 NUMBER OF FDA ORPHAN DRUG DESIGNATIONS AND APPROVALS PER
YEAR, 1983-2012 14
ORPHAN DRUGS: GLOBAL STATUS 14
EUROPEAN UNION 15
AUSTRALIA 16
SINGAPORE 16
JAPAN 17
TAIWAN 17
CANADA 18
BRAZIL 18
SOUTH KOREA 18
ISRAEL 18
TABLE 4 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU,
AUSTRALIA AND JAPAN 19
CLASSIFICATION OF ORPHAN DRUGS 20
BIOLOGICAL PRODUCTS 20
NONBIOLOGICAL PRODUCTS 20
TABLE 5 BIOLOGICAL ORPHAN DRUGS BY TRADE NAME AND INDICATION 21
TABLE 6 NONBIOLOGICAL ORPHAN DRUGS BY TRADE NAME AND INDICATION 22
TABLE 7 EXAMPLES OF ORPHAN DRUGS, INDICATIONS AND PREVALENCE OF
DISEASES 27
INDUSTRY OUTLOOK TOWARD ORPHAN DRUGS 27
MARKET EXCLUSIVITY 28
PRICING AND REGULATORY RESTRICTIONS ON PROFITABILITY 28
OFF-LABEL USE 28
FUTURE CHALLENGES FOR THE ODA 29
COMPANIES WITH ORPHAN DRUG APPROVALS 29
TABLE 8 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG APPROVALS,*
1983-2012 30
ORPHAN DRUG ACCESS AND REIMBURSEMENT 31
UNITED STATES 31
EUROPE 32
CHAPTER 4 NEW PRODUCT DEVELOPMENT AND REGULATION OF ORPHAN DRUGS 34
INTRODUCTION 34
PROCESS AND APPROVAL OF ORPHAN DRUGS 35
RECENT ORPHAN DRUG APPROVALS 35
Kalydeco (Ivacaftor) 35
Carbaglu (Carglumic Acid) 35
Vpriv (Velaglucerase) 36
Pomalyst (Pomalidomide) 36
Abthrax (Raxibacumab) 36
Corifact (Factor XIII Concentrate [Human]) 36
Vpriv (Velaglucerase-Alfa Injection) 36
TABLE 9 ORPHAN DRUG MARKETING APPROVALS, 2008 THROUGH FEB. 2013 37
RECALLS AND SAFETY ALERTS 52
TABLE 10 ORPHAN DRUG SAFETY ALERTS AND RECALLS, 2008-2013 52
CHAPTER 5 GLOBAL MARKET FOR ORPHAN DRUGS 56
MARKET BY TYPE 56
MARKET OVERVIEW 57
Recent Orphan Drug Approvals 58
Nplate (Romiplostim) 58
Nulojix (Belatacept) 58
Avastin (Bevacizumab) 58
Sensipar (Cinacalcet) 58
Neupogen/Imumax (filgrastim) and Neutroval (Tbo-filgrastim) 59
Gammagard Liquid (Immune Globulin Infusion [Human]) 59
Multiferon (Interferon Alpha) 59
Avonex/Rebif (Interferon Beta-1a) 59
Rituxan/MabThera (Rituximab) 59
Herclon/Herceptin (Trastuzumab) 60
VariZIG (Varicella Zoster Immune Globulin) 60
Afinitor (Everolimus) 60
Sirturo (Bedaquiline) 60
Vidaza (Azacitidine) 61
Onfi (Clobazam) 61
NovoSeven RT (rFVIIa) 61
Sprycel (Dasatinib) 61
Gleevec/Glivec (Imatinib Mesylate) 61
Cerezyme (Imiglucerase) 62
Thalomid (Thalidomide) 62
Tasigna (Nilotinib) 62
Temodar (Temozolomide) 62
Thyrogen (Thyrotropin Alfa for Injection) 62
TOBI Podhaler (Tobramycin) 63
Topamax (Topiramate) 63
Zometa/Zomera/Aclasta/Reclast (Zoledronic or Zoledronate) 63
MARKET REVENUE 63
TABLE 11 GLOBAL SALES OF ORPHAN DRUGS BY TYPE, THROUGH 2017 ($
MILLIONS) 65
FIGURE 1 GLOBAL SALES OF ORPHAN DRUGS BY TYPE, 2010-2017 ($ MILLIONS) 65
MARKET SHARES 65
TABLE 12 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 66
FIGURE 2 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 66
MARKET BY REGION 66
MARKET OVERVIEW 67
MARKET REVENUE 68
TABLE 13 GLOBAL SALES OF ORPHAN DRUGS BY REGION, THROUGH 2017 ($
MILLIONS) 69
FIGURE 3 GLOBAL SALES OF ORPHAN DRUGS BY REGION, 2010-2017 ($ MILLIONS) 69
MARKET SHARES 69
TABLE 14 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 70
FIGURE 4 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 70
Market for Biological Orphan Drugs by Region 70
TABLE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 71
FIGURE 5 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017
($ MILLIONS) 72
Market Shares 72
TABLE 16 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 72
FIGURE 6 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 72
Market for Nonbiological Orphan Drugs by Region 73
TABLE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 74
FIGURE 7 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 75
Market Shares 75
TABLE 18 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 75
FIGURE 8 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 75
MARKETS BY END USERS 76
MARKET OVERVIEW 76
Nephrology and Other Infectious Diseases 76
Oncology 77
Chronic Lymphocytic Leukemia 78
HER2-Positive Metastatic Cancer 78
Advanced Follicular Lymphoma 78
Metastatic Breast Cancer 79
HR+ and HER2/HER2- Advanced Breast Cancer 79
Metastatic Colorectal Cancer 79
Metastatic Melanoma 79
Metastatic Non-small Cell Lung Cancer 80
Philadelphia Chromosome-Positive (Ph+) Chronic Myelogenous
Leukemia 80
Pancreatic Neuroendocrine Tumors 81
PUA Levels in Patients with Leukemia, Lymphoma and Solid
Tumor Malignancies 81
Neurological Disorders 82
Multiple Sclerosis 82
Lou Gehrig's Disease 82
Alzheimer's Disease 82
Multifocal Motor Neuropathy 82
Stem Cell Transplantation 82
Other Rare Diseases 83
Chronic Immune (Idiopathic) Thrombocytopenic Purpura 83
ANCA-Associated Vasculitis 84
Fabry Disease 84
Varicella Zoster Virus 84
Gouty Arthritis 85
Non-Transfusion-Dependent Thalassemia/Beta-Thalassemia
Major 85
Cushing's Syndrome 86
MARKET REVENUE 86
TABLE 19 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
THROUGH 2017 ($ MILLIONS) 87
FIGURE 9 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
2010-2017 ($ MILLIONS) 87
MARKET SHARES 88
TABLE 20 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 88
FIGURE 10 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 89
CHAPTER 6 MARKET ANALYSIS OF BIOLOGICAL ORPHAN DRUGS 91
MARKET FOR BIOLOGICAL ORPHAN DRUGS BY TYPE 92
MARKET OVERVIEW 92
Nplate (Romiplostim) 92
Fabrazyme (Agalsidase Beta) 93
Lumizyme/Myozyme (Alglucosidase Alfa) 93
Nulojix (Belatacept) 93
Avastin (Bevacizumab) 93
Ilaris (Canakinumab) 93
Sensipar (Cinacalcet) 93
Epogen/Procrit (Epoetin Alfa) 94
Enbrel (Etanercept) 94
Neupogen/Imumax (Filgrastim) and Neutroval (Tbo-filgrastim) 94
Gammagard Liquid (Immune Globulin Infusion [Human]) 94
Remicade (Infliximab) 94
Multiferon (Interferon Alpha) 95
Avonex/Rebif (Interferon Beta-1a) 95
Yervoy (Ipilimumab) 95
Rituxan/MabThera (Rituximab) 95
Herclon/Herceptin (Trastuzumab) 96
VariZIG (Varicella Zoster Immune Globulin) 96
MARKET REVENUE 96
Fabrazyme (Agalsidase Beta)/Myozyme (Alglucosidase Alfa) 96
Nulojix (Belatacept) 96
Avastin (Bevacizumab) 97
Ilaris (Canakinumab) 97
Sensipar (Cinacalcet) 97
Epogen (Epoetin Alfa) 97
Enbrel (Etanercept) 97
Neupogen/Imumax (filgrastim) and Neutroval (Tbo-filgrastim) 97
Immune Globulin Infusions 98
Remicade (Infliximab) 98
Interferon Alfa and Interferon Beta 98
Yervoy (Ipilimumab) 98
Ribavirin USP and Rituxan (Rituximab) 98
VariZIG (Varicella Zoster Immune Globulin [Human]) 99
Nplate (Romiplostim) 99
Herclon/Herceptin (Trastuzumab) 99
"Others" Segment 99
TABLE 21 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY ACTIVE INGREDIENT,
THROUGH 2017 ($ MILLIONS) 100
FIGURE 11 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY ACTIVE
INGREDIENT, 2010-2017 ($ MILLIONS) 100
MARKET SHARES 101
TABLE 22 GLOBAL MARKET SHARES OF SALES OF MAJOR BIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
FIGURE 12 GLOBAL MARKET SHARES OF SALES OF MAJOR BIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 103
MARKET BY REGION 103
MARKET OVERVIEW 104
MARKET REVENUE 105
TABLE 23 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 106
FIGURE 13 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017
($ MILLIONS) 106
MARKET SHARES 106
TABLE 24 GLOBAL MARKET SHARES OF SALES OF BIOLOGICAL ORPHAN DRUGS BY
REGION, 2011 (%) 107
FIGURE 14 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY REGION,
2011 (%) 107
MARKETS BY APPLICATIONS 107
TABLE 25 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF MEDICAL
APPLICATION, THROUGH 2017 ($ MILLIONS) 108
FIGURE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF MEDICAL
APPLICATION, 2010-2017 ($ MILLIONS) 108
NEPHROLOGY AND INFECTIOUS DISEASES 108
Nulojix (Belatacept) 109
Sensipar/Mimpara (Cinacalcet) 109
Nplate (Romiplostim) 109
Market Revenue 110
TABLE 26 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEPHROLOGY
AND TO TREAT INFECTIOUS DISEASES BY ACTIVE INGREDIENT, THROUGH 2017 ($
MILLIONS)
110
FIGURE 16 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEPHROLOGY
AND TO TREAT INFECTIOUS DISEASES BY ACTIVE INGREDIENT, 2010-2017 ($
MILLIONS)
110
NEUROLOGY 111
Avonex/Avonex Pen (Interferon Beta) 111
Gammagard Liquid 10%/Kiovig [Immune Globulin Infusion (Human)] 112
Market Revenue 112
TABLE 27 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEUROLOGY
BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 113
FIGURE 17 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEUROLOGY
BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 113
ONCOLOGY 113
Neulasta (Pegfilgrastim)/Neupogen (Filgrastim) 113
Elitek (Rasburicase) 114
Nplate (Romiplostim) 114
Rituxan (Rituximab) 115
Herceptin (Trastuzumab) 115
Yervoy (Ipilimumab) 115
Xgeva (Denosumab) 116
Avastin (Bevacizumab) 116
Market Revenue 116
TABLE 28 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN ONCOLOGY BY
ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 117
FIGURE 18 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN ONCOLOGY
BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 118
MARKET FOR DRUGS USED IN THE "OTHERS" SEGMENT 118
Lumizyme/Myozyme (Alglucosidase Alfa) 118
Rituxan (Rituximab) 119
Fabrazyme (Agalsidase Beta) 119
VariZIG [Varicella Zoster Immune Globulin (Human)] 119
MARKET REVENUE 120
TABLE 29 GLOBAL SALES OF BIOLOGICAL DRUGS USED TO TREAT OTHER
DISEASES BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 120
FIGURE 19 GLOBAL SALES OF BIOLOGICAL DRUGS USED TO TREAT OTHER
DISEASES BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 121
MARKET SHARES 121
TABLE 30 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2011 (%) 121
FIGURE 20 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2011 (%) 121
INDUSTRY STRUCTURE 122
MARKET LEADERS 122
TABLE 31 MAJOR MANUFACTURERS OF BIOLOGICAL ORPHAN DRUGS 122
MARKET SHARES 123
TABLE 32 MANUFACTURER SHARES OF THE GLOBAL MARKET FOR BIOLOGICAL
ORPHAN DRUGS, 2011 (%) 123
FIGURE 21 MANUFACTURER SHARES OF THE MARKET FOR BIOLOGICAL ORPHAN
DRUGS, 2011 (%) 124
CHAPTER 7 PATENT ANALYSIS 126
PATENTS BY YEAR 126
TABLE 33 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 127
FIGURE 22 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 127
PATENTS BY CATEGORY 127
TABLE 34 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 127
FIGURE 23 NUMBER OF PATENTS FOR U.S. ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 128
PATENTS BY COUNTRY 128
TABLE 35 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY,
2008 TO FEB. 2013 128
FIGURE 24 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY,
2008 TO FEB. 2013 129
TABLE 36 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008 TO
FEB. 2013 (NO. OF PATENTS/%) 129
FIGURE 25 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008
TO FEB. 2013 (%) 129
PATENTS BY COMPANY 130
TABLE 37 U.S. ORPHAN DRUG PATENTS BY COMPANY, 2008 TO FEB. 2013 (NO.) 130
PATENTS BY ASSIGNEE 131
TABLE 38 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 (NO.) 131
FIGURE 26 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE,
2008 TO FEB. 2013 131
CHAPTER 8 CURRENT SITUATION 134
CHAPTER 9 COMPANY PROFILES 139
ACORDA THERAPEUTICS 139
AEGERION PHARMACEUTICALS 139
ALEXION PHARMACEUTICALS 139
AMGEN INC. 140
ARIAD PHARMACEUTICALS 140
ASTRAZENECA PHARMACEUTICAL LP 141
BAXTER INTERNATIONAL 141
BAYER SCHERING 141
BRISTOL-MYERS SQUIBB 143
CANGENE BIOPHARMA 144
CELGENE CORP. 144
EXELIXIS 145
GENENTECH INC. 146
GENZYME 146
GLAXOSMITHKLINE 147
HYPERION THERAPEUTICS 148
JANSSEN RESEARCH and DEVELOPMENT, LLC 149
JOHNSON and JOHNSON 149
LUNDBECK 150
MERCK 150
NOVARTIS PHARMACEUTICALS 151
NOVO NORDISK 153
NPS PHARMACEUTICALS 153
ONYX THERAPEUTICS 154
PFIZER 154
RARE DISEASE THERAPEUTICS 155
ROCHE 156
SANOFI 157
SEATTLE GENETICS 158
SHIRE ORPHAN THERAPIES 158
SIGMA-TAU PHARMACEUTICALS INC. 159
SWEDISH ORPHAN BIOVITRUM AB (SOBI) 159
TALON THERAPEUTICS 159

LIST OF TABLES
SUMMARY TABLE GLOBAL SALES OF ORPHAN DRUGS BY TYPE, THROUGH 2017 ($
MILLIONS) 8
TABLE 1 HISTORY OF ORPHAN DRUG LEGISLATION 11
TABLE 2 U.S. ORPHAN DRUG ACT, AMENDMENTS AND OTHER LEGISLATION 13
TABLE 3 NUMBER OF FDA ORPHAN DRUG DESIGNATIONS AND APPROVALS PER
YEAR, 1983-2012 14
TABLE 4 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU, AUSTRALIA
AND JAPAN 19
TABLE 5 BIOLOGICAL ORPHAN DRUGS BY TRADE NAME AND INDICATION 21
TABLE 6 NONBIOLOGICAL ORPHAN DRUGS BY TRADE NAME AND INDICATION 22
TABLE 7 EXAMPLES OF ORPHAN DRUGS, INDICATIONS AND PREVALENCE OF
DISEASES 27
TABLE 8 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG APPROVALS,*
1983-2012 30
TABLE 9 ORPHAN DRUG MARKETING APPROVALS, 2008 THROUGH FEB. 2013 37
TABLE 10 ORPHAN DRUG SAFETY ALERTS AND RECALLS, 2008-2013 52
TABLE 11 GLOBAL SALES OF ORPHAN DRUGS BY TYPE, THROUGH 2017 ($ MILLIONS) 65
TABLE 12 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 66
TABLE 13 GLOBAL SALES OF ORPHAN DRUGS BY REGION, THROUGH 2017 ($
MILLIONS) 69
TABLE 14 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 70
TABLE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 71
TABLE 16 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 72
TABLE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 74
TABLE 18 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 75
TABLE 19 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
THROUGH 2017 ($ MILLIONS) 87
TABLE 20 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF MEDICAL
APPLICATION, 2011 (%) 88
TABLE 21 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY ACTIVE INGREDIENT,
THROUGH 2017 ($ MILLIONS) 100
TABLE 22 GLOBAL MARKET SHARES OF SALES OF MAJOR BIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
TABLE 23 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 106
TABLE 24 GLOBAL MARKET SHARES OF SALES OF BIOLOGICAL ORPHAN DRUGS BY
REGION, 2011 (%) 107
TABLE 25 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF MEDICAL
APPLICATION, THROUGH 2017 ($ MILLIONS) 108
TABLE 26 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEPHROLOGY
AND TO TREAT INFECTIOUS DISEASES BY ACTIVE INGREDIENT, THROUGH 2017 ($
MILLIONS) 110
TABLE 27 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEUROLOGY BY
ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 113
TABLE 28 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN ONCOLOGY BY
ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 117
TABLE 29 GLOBAL SALES OF BIOLOGICAL DRUGS USED TO TREAT OTHER DISEASES
BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 120
TABLE 30 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2011 (%) 121
TABLE 31 MAJOR MANUFACTURERS OF BIOLOGICAL ORPHAN DRUGS 122
TABLE 32 MANUFACTURER SHARES OF THE GLOBAL MARKET FOR BIOLOGICAL
ORPHAN DRUGS, 2011 (%) 123
TABLE 33 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 127
TABLE 34 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 127
TABLE 35 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY, 2008
TO FEB. 2013 128
TABLE 36 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008 TO
FEB. 2013 (NO. OF PATENTS/%) 129
TABLE 37 U.S. ORPHAN DRUG PATENTS BY COMPANY, 2008 TO FEB. 2013 (NO.) 130
TABLE 38 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 (NO.) 131

LIST OF FIGURES
SUMMARY FIGURE GLOBAL SALES OF ORPHAN DRUGS BY TYPE, 2010-2017 ($
MILLIONS) 8
FIGURE 1 GLOBAL SALES OF ORPHAN DRUGS BY TYPE, 2010-2017 ($ MILLIONS) 65
FIGURE 2 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 66
FIGURE 3 GLOBAL SALES OF ORPHAN DRUGS BY REGION, 2010-2017 ($ MILLIONS) 69
FIGURE 4 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 70
FIGURE 5 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017 ($
MILLIONS) 72
FIGURE 6 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 72
FIGURE 7 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 75
FIGURE 8 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 75
FIGURE 9 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
2010-2017 ($ MILLIONS) 87
FIGURE 10 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 89
FIGURE 11 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY ACTIVE INGREDIENT,
2010-2017 ($ MILLIONS) 100
FIGURE 12 GLOBAL MARKET SHARES OF SALES OF MAJOR BIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 103
FIGURE 13 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017
($ MILLIONS) 106
FIGURE 14 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY REGION,
2011 (%) 107
FIGURE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF MEDICAL
APPLICATION, 2010-2017 ($ MILLIONS) 108
FIGURE 16 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEPHROLOGY
AND TO TREAT INFECTIOUS DISEASES BY ACTIVE INGREDIENT, 2010-2017 ($
MILLIONS)
110
FIGURE 17 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN NEUROLOGY BY
ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 113
FIGURE 18 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS USED IN ONCOLOGY BY
ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 118
FIGURE 19 GLOBAL SALES OF BIOLOGICAL DRUGS USED TO TREAT OTHER
DISEASES BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 121
FIGURE 20 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2011 (%) 121
FIGURE 21 MANUFACTURER SHARES OF THE MARKET FOR BIOLOGICAL ORPHAN
DRUGS, 2011 (%) 124
FIGURE 22 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 127
FIGURE 23 NUMBER OF PATENTS FOR U.S. ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 128
FIGURE 24 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY,
2008 TO FEB. 2013 129
FIGURE 25 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008 TO
FEB. 2013 (%) 129
FIGURE 26 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 131

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