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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.

CONTRIBUTIONS OF THE STUDY AND INTENDED AUDIENCE

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 Non-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 2
METHODOLOGY 3
INFORMATION SOURCES 3
ABOUT THE AUTHOR 3
RELATED BCC RESEARCH REPORTS 3
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) 58
Gammagard Liquid (Immune Globulin Infusion [Human]) 59
Multiferon (Interferon Alpha) 59
Avonex/Rebif (Interferon Beta-1a) 59
Rituxan/MabThera (Rituximab) 59
Herclon/Herceptin (Trastuzumab) 59
VariZIG (Varicella Zoster Immune Globulin) 60
Afinitor (Everolimus) 60
Sirturo (Bedaquiline) 60
Vidaza (Azacitidine) 60
Onfi (Clobazam) 61
NovoSeven RT (rFVIIa) 61
Sprycel (Dasatinib) 61
Gleevec/Glivec (Imatinib Mesylate) 61
Cerezyme (Imiglucerase) 61
Thalomid (Thalidomide) 62
Tasigna (Nilotinib) 62
Temodar (Temozolomide) 62
Thyrogen (Thyrotropin Alfa for Injection) 62
TOBI Podhaler (Tobramycin) 62
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) 64
FIGURE 1 GLOBAL SALES OF ORPHAN DRUGS BY TYPE, 2010-2017 ($ MILLIONS) 64
MARKET SHARES 64
TABLE 12 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 65
FIGURE 2 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 65
MARKET BY REGION 65
MARKET OVERVIEW 66
MARKET REVENUE 67
TABLE 13 GLOBAL SALES OF ORPHAN DRUGS BY REGION, THROUGH 2017 ($
MILLIONS) 68
FIGURE 3 GLOBAL SALES OF ORPHAN DRUGS BY REGION, 2010-2017 ($ MILLIONS) 68
MARKET SHARES 68
TABLE 14 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 69
FIGURE 4 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 69
Market for Biological Orphan Drugs by Region 69
TABLE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 70
FIGURE 5 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017
($ MILLIONS) 71
Market Shares 71
TABLE 16 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 71
FIGURE 6 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 71
Market for Nonbiological Orphan Drugs by Region 72
TABLE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 73
FIGURE 7 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 74
Market Shares 74
TABLE 18 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 74
FIGURE 8 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 74
MARKETS BY END USERS 75
MARKET OVERVIEW 75
Nephrology and Other Infectious Diseases 75
Oncology 76
Chronic Lymphocytic Leukemia 77
HER2-Positive Metastatic Cancer 77
Advanced Follicular Lymphoma 77
Metastatic Breast Cancer 78
HR+ and HER2/HER2- Advanced Breast Cancer 78
Metastatic Colorectal Cancer 78
Metastatic Melanoma 78
Metastatic Non-small Cell Lung Cancer 79
Philadelphia Chromosome-Positive (Ph+) Chronic Myelogenous
Leukemia 79
Pancreatic Neuroendocrine Tumors 80
PUA Levels in Patients with Leukemia, Lymphoma and Solid
Tumor Malignancies 80
Neurological Disorders 81
Multiple Sclerosis 81
Lou Gehrig's Disease 81
Alzheimer's Disease 81
Multifocal Motor Neuropathy 81
Stem Cell Transplantation 81
Other Rare Diseases 82
Chronic Immune (Idiopathic) Thrombocytopenic Purpura 82
ANCA-Associated Vasculitis 83
Fabry Disease 83
Varicella Zoster Virus 83
Gouty Arthritis 84
Non-Transfusion-Dependent Thalassemia/Beta-Thalassemia
Major 84
Cushing's Syndrome 85
MARKET REVENUE 85
TABLE 19 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
THROUGH 2017 ($ MILLIONS) 86
FIGURE 9 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
2010-2017 ($ MILLIONS) 86
MARKET SHARES 87
TABLE 20 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 87
FIGURE 10 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 88
CHAPTER 6 MARKET ANALYSIS OF NONBIOLOGICAL ORPHAN DRUGS 90
MARKET FOR NONBIOLOGICAL ORPHAN DRUGS 90
MARKET OVERVIEW 90
Recently Approved Nonbiological Orphan Drugs 91
Afinitor (Everolimus) 91
Sirturo (Bedaquiline) 91
Cystaran (Cysteamine Ophthalmic Solution) 91
Vidaza (Azacitidine) 91
Onfi (Clobazam) 92
NovoSeven (Coagulation Factor VIIa Recombinant) 92
Xalkori (Crizotinib) 92
Votrient (Pazopanib) 92
Ampyra/Fampyra (Dalfampridine) 92
Sprycel (Dasatinib) 93
Exjade (Deferasirox) 93
Zortress/Afinitor/Certican (Everolimus) 93
Aromasin (Exemestane) 93
Gleevec/Glivec (Imatinib Mesylate) 94
Cerezyme (Imiglucerase) 94
Lamictal (Lamotrigine) 94
Thalomid (Thalidomide) 94
Revlimid (Lenalidomide) 94
Tasigna (Nilotinib) 95
Istodax (Romidepsin) 95
Somatropin Recombinant Human Growth Hormone 95
Nexavar (Sorafenib) 95
Temodar (Temozolomide) 96
Thyrogen (Thyrotropin Alfa for Injection) 96
TOBI Podhaler (Tobramycin) 96
Zometa/Zomera/Aclasta/Reclast (Zoledronic or Zoledronate) 96
MARKET REVENUE 97
Vidaza (Azacitidine) 97
NovoSeven (Coagulation Factor VIIa Recombinant) 97
Onfi (Clobazam) and Xalkori (Crizotinib) 97
Dalfampridine and Deferasirox 97
Sprycel (Dasatinib) 97
Exjade (Deferasirox) 97
Gleevec/Glivec (Imatinib Mesylate) 98
Afinitor (Everolimus) 98
Aromasin (Exemestane) 98
Cerezyme (Imiglucerase) 98
Revlimid (Lenalidomide) 98
Votrient (Pazopanib) 98
Lamictal (Lamotrigine) 98
Tasigna (Nilotinib) 99
Pazopanib 99
Romidepsin 99
Somatropin and Sorafenib 99
Topamax (Topiramate) 99
TOBI Podhaler (Tobramycin) 99
Thalomid (Thalidomide) 99
Temozolomide 100
Zometa (Zoledronic) 100
Thyrogen (Thyrotropin Alfa) 100
TABLE 21 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY ACTIVE
INGREDIENT, THROUGH 2017 ($ MILLIONS) 100
FIGURE 11 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY ACTIVE
INGREDIENT, 2010-2017 ($ MILLIONS) 101
MARKET SHARES 102
TABLE 22 GLOBAL MARKET SHARES OF SALES OF MAJOR NONBIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
FIGURE 12 GLOBAL MARKET SHARES OF SALES OF MAJOR NONBIOLOGICAL
ORPHAN DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
MARKET BY REGION 103
MARKET OVERVIEW 104
MARKET REVENUE 106
TABLE 23 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 106
FIGURE 13 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 106
MARKET SHARES 107
TABLE 24 GLOBAL MARKET SHARES OF SALES OF NONBIOLOGICAL ORPHAN
DRUGS BY REGION, 2011 (%) 107
FIGURE 14 GLOBAL MARKET SHARES OF SALES OF NONBIOLOGICAL ORPHAN
DRUGS BY REGION, 2011 (%) 107
MARKET BY AREA OF MEDICAL APPLICATION 107
TABLE 25 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, THROUGH 2017 ($ MILLIONS) 108
FIGURE 15 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2010-2017 ($ MILLIONS) 108
BLOOD RELATED DISORDERS AND CYSTIC FIBROSIS 108
Ilaris (Canakinumab) 109
Exjade (Deferasirox) 109
Market Revenues 110
TABLE 26 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED TO TREAT
BLOOD RELATED DISORDERS AND CYSTIC FIBROSIS BY ACTIVE INGREDIENT,
THROUGH 2017 ($ MILLIONS)
111
FIGURE 16 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED TO TREAT
BLOOD RELATED DISORDERS AND CYSTIC FIBROSIS BY ACTIVE INGREDIENT,
2010-2017 ($ MILLIONS)
111
NEUROLOGY 111
Market Revenue 112
TABLE 27 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
NEUROLOGY BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 112
FIGURE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
NEUROLOGY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 112
ONCOLOGY 113
Market Revenue 116
TABLE 28 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
ONCOLOGY BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 117
FIGURE 18 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
ONCOLOGY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 117
OTHERS 118
Market Revenue 119
TABLE 29 GLOBAL SALES OF NONBIOLOGICAL DRUGS USED TO TREAT DISEASES
IN THE “OTHERS” CATEGORY BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 120
FIGURE 19 GLOBAL SALES OF NONBIOLOGICAL DRUGS USED TO TREAT DISEASES
IN THE "OTHERS" CATEGORY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 120
MARKET SHARES 120
TABLE 30 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA
OF MEDICAL APPLICATION, 2011 (%) 121
FIGURE 20 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUGS BY
AREA OF MEDICAL APPLICATION, 2011 (%) 121
INDUSTRY STRUCTURE 121
MARKET LEADERS 121
TABLE 31 MAJOR MANUFACTURERS OF NONBIOLOGICAL ORPHAN DRUGS 122
MARKET SHARES 122
TABLE 32 MANUFACTURER SHARES OF THE MARKET FOR NONBIOLOGICAL
ORPHAN DRUGS, 2011 (%) 123
FIGURE 21 MANUFACTURER SHARES OF THE MARKET FOR NONBIOLOGICAL
ORPHAN DRUGS, 2011 (%) 123
CHAPTER 7 PATENT ANALYSIS 125
PATENTS BY YEAR 125
TABLE 33 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 126
FIGURE 22 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 126
PATENTS BY CATEGORY 126
TABLE 34 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 126
FIGURE 23 NUMBER OF PATENTS FOR U.S. ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 127
PATENTS BY COUNTRY 127
TABLE 35 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY,
2008 TO FEB. 2013 127
FIGURE 24 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/%) 128
FIGURE 25 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008
TO FEB. 2013 (%) 128
PATENTS BY COMPANY 129
TABLE 37 U.S. ORPHAN DRUG PATENTS BY COMPANY, 2008 TO FEB. 2013 (NO.) 129
PATENTS BY ASSIGNEE 130
TABLE 38 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 (NO.) 130
FIGURE 26 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE,
2008 TO FEB. 2013 130
CHAPTER 8 CURRENT SITUATION 133
CHAPTER 9 COMPANY PROFILES 138
ACORDA THERAPEUTICS 138
AEGERION PHARMACEUTICALS 138
ALEXION PHARMACEUTICALS 138
AMGEN INC. 139
ARIAD PHARMACEUTICALS 139
ASTRAZENECA PHARMACEUTICAL LP 140
BAXTER INTERNATIONAL 140
BAYER SCHERING 140
BRISTOL-MYERS SQUIBB 142
CANGENE BIOPHARMA 143
CELGENE CORP. 143
EXELIXIS 144
GENENTECH INC. 145
GENZYME 145
GLAXOSMITHKLINE 146
HYPERION THERAPEUTICS 147
JANSSEN RESEARCH and DEVELOPMENT, LLC 148
JOHNSON and JOHNSON 148
LUNDBECK 149
MERCK 149
NOVARTIS PHARMACEUTICALS 150
NOVO NORDISK 152
NPS PHARMACEUTICALS 152
ONYX THERAPEUTICS 153
PFIZER 153
RARE DISEASE THERAPEUTICS 154
ROCHE 155
SANOFI 156
SEATTLE GENETICS 157
SHIRE ORPHAN THERAPIES 157
SIGMA-TAU PHARMACEUTICALS INC. 158
SWEDISH ORPHAN BIOVITRUM AB (SOBI) 158
TALON THERAPEUTICS 158

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) 64
TABLE 12 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 65
TABLE 13 GLOBAL SALES OF ORPHAN DRUGS BY REGION, THROUGH 2017 ($
MILLIONS) 68
TABLE 14 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 69
TABLE 15 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH
2017 ($ MILLIONS) 70
TABLE 16 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 71
TABLE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 73
TABLE 18 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 74
TABLE 19 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
THROUGH 2017 ($ MILLIONS) 86
TABLE 20 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF MEDICAL
APPLICATION, 2011 (%) 87
TABLE 21 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY ACTIVE
INGREDIENT, THROUGH 2017 ($ MILLIONS) 100
TABLE 22 GLOBAL MARKET SHARES OF SALES OF MAJOR NONBIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
TABLE 23 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
THROUGH 2017 ($ MILLIONS) 106
TABLE 24 GLOBAL MARKET SHARES OF SALES OF NONBIOLOGICAL ORPHAN DRUGS
BY REGION, 2011 (%) 107
TABLE 25 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, THROUGH 2017 ($ MILLIONS) 108
TABLE 26 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED TO TREAT
BLOOD RELATED DISORDERS AND CYSTIC FIBROSIS BY ACTIVE INGREDIENT,
THROUGH 2017 ($ MILLIONS)
111
TABLE 27 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
NEUROLOGY BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 112
TABLE 28 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN ONCOLOGY
BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 117
TABLE 29 GLOBAL SALES OF NONBIOLOGICAL DRUGS USED TO TREAT DISEASES IN
THE “OTHERS” CATEGORY BY ACTIVE INGREDIENT, THROUGH 2017 ($ MILLIONS) 120
TABLE 30 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA
OF MEDICAL APPLICATION, 2011 (%) 121
TABLE 31 MAJOR MANUFACTURERS OF NONBIOLOGICAL ORPHAN DRUGS 122
TABLE 32 MANUFACTURER SHARES OF THE MARKET FOR NONBIOLOGICAL ORPHAN
DRUGS, 2011 (%) 123
TABLE 33 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 126
TABLE 34 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 126
TABLE 35 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY, 2008
TO FEB. 2013 127
TABLE 36 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008 TO
FEB. 2013 (NO. OF PATENTS/%) 128
TABLE 37 U.S. ORPHAN DRUG PATENTS BY COMPANY, 2008 TO FEB. 2013 (NO.) 129
TABLE 38 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 (NO.) 130

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) 64
FIGURE 2 GLOBAL MARKET SHARES OF MAJOR ORPHAN DRUGS BY TYPE, 2011 (%) 65
FIGURE 3 GLOBAL SALES OF ORPHAN DRUGS BY REGION, 2010-2017 ($ MILLIONS) 68
FIGURE 4 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY REGION, 2011 (%) 69
FIGURE 5 GLOBAL SALES OF BIOLOGICAL ORPHAN DRUGS BY REGION, 2010-2017 ($
MILLIONS) 71
FIGURE 6 GLOBAL MARKET SHARES OF BIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 71
FIGURE 7 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 74
FIGURE 8 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUG SALES BY
REGION, 2011 (%) 74
FIGURE 9 GLOBAL SALES OF ORPHAN DRUGS BY AREA OF MEDICAL APPLICATION,
2010-2017 ($ MILLIONS) 86
FIGURE 10 GLOBAL MARKET SHARES OF ORPHAN DRUG SALES BY AREA OF
MEDICAL APPLICATION, 2011 (%) 88
FIGURE 11 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY ACTIVE
INGREDIENT, 2010-2017 ($ MILLIONS) 101
FIGURE 12 GLOBAL MARKET SHARES OF SALES OF MAJOR NONBIOLOGICAL ORPHAN
DRUGS BY ACTIVE INGREDIENT, 2011 (%) 102
FIGURE 13 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY REGION,
2010-2017 ($ MILLIONS) 106
FIGURE 14 GLOBAL MARKET SHARES OF SALES OF NONBIOLOGICAL ORPHAN
DRUGS BY REGION, 2011 (%) 107
FIGURE 15 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA OF
MEDICAL APPLICATION, 2010-2017 ($ MILLIONS) 108
FIGURE 16 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED TO TREAT
BLOOD RELATED DISORDERS AND CYSTIC FIBROSIS BY ACTIVE INGREDIENT,
2010-2017 ($ MILLIONS)
111
FIGURE 17 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
NEUROLOGY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 112
FIGURE 18 GLOBAL SALES OF NONBIOLOGICAL ORPHAN DRUGS USED IN
ONCOLOGY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 117
FIGURE 19 GLOBAL SALES OF NONBIOLOGICAL DRUGS USED TO TREAT DISEASES IN
THE "OTHERS" CATEGORY BY ACTIVE INGREDIENT, 2010-2017 ($ MILLIONS) 120
FIGURE 20 GLOBAL MARKET SHARES OF NONBIOLOGICAL ORPHAN DRUGS BY AREA
OF MEDICAL APPLICATION, 2011 (%) 121
FIGURE 21 MANUFACTURER SHARES OF THE MARKET FOR NONBIOLOGICAL ORPHAN
DRUGS, 2011 (%) 123
FIGURE 22 NUMBER OF U.S. PATENTS FOR ORPHAN DRUGS BY YEAR, 2008 TO FEB.
2013 126
FIGURE 23 NUMBER OF PATENTS FOR U.S. ORPHAN DRUGS BY MAJOR CATEGORY,
2008 TO FEB. 2013 127
FIGURE 24 NUMBER OF U.S. ORPHAN DRUG PATENTS BY ASSIGNEE'S COUNTRY,
2008 TO FEB. 2013 128
FIGURE 25 U.S. ORPHAN DRUG PATENT SHARES BY ASSIGNEE'S COUNTRY, 2008 TO
FEB. 2013 (%) 128
FIGURE 26 U.S. PATENTS ISSUED FOR ORPHAN DRUGS BY TYPE OF ASSIGNEE, 2008
TO FEB. 2013 130

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