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Global Markets for Orphan Drugs

Global Markets for Orphan Drugs

The global orphan drug market totaled nearly $123 billion in 2014 and will continue to grow to reach nearly $191 billion by 2019, demonstrating a compound annual growth rate (CAGR) of 9.2% during the forecast period (2014 to 2019).

This report provides:

An overview of orphan drug criteria, the history of the Orphan Drug Act and its influence, advantages inherent in orphan drug designation, and future growth trends.

Analyses of global market trends, with data from 2013 and 2014, and projections of compound annual growth rates (CAGRs) through 2019.

Detailed discussion of successful orphan products.

Detailed analysis of blockbuster orphan drugs.

The rising influence of pharmacogenomics in the orphan drug market.

A discussion of specific rare diseases for which orphan drugs exist.

Analysis of the most promising therapeutic areas in the industry and leading orphan drugs in these areas.

An outline of legislation and regulation, including public and private agencies involved in support and development.

Technology trends analysis within the orphan drug market.

Comprehensive company profiles of major players in the industry.


SCOPE OF THE REPORT

The scope of this study includes orphan drugs used in pharmaceutical markets globally. This report analyzes the therapeutic applications of orphan drugs in rare and orphan diseases as well as regulatory framework, patents, and recent innovations. The report also analyzes each submarket and its applications, projections and market shares. Technological issues include the latest trends and developments.

This report covers U.S. and European markets as well as emerging global markets like India, Japan, China, Singapore, Taiwan, Korea, Canada, Brazil, Africa, Australia, New Zealand, etc.

CHAPTER 1 INTRODUCTION
STUDY OBJECTIVES
REASONS FOR DOING THIS REPORT
CONTRIBUTIONS OF THE REPORT AND FOR WHOM
SCOPE OF THE REPORT
METHODOLOGY
INFORMATION SOURCES
ANALYSTS' CREDENTIALS
RELATED BCC RESEARCH REPORTS
BCC RESEARCH WEBSITE
DISCLAIMER
CHAPTER 2 EXECUTIVE SUMMARY
SUMMARY TABLE GLOBAL REVENUES OF ORPHAN DRUGS BY TYPE, THROUGH 2019 ($ MILLIONS)
SUMMARY FIGURE GLOBAL REVENUES OF ORPHAN DRUGS BY TYPE, 2012-2019 ($ MILLIONS)
CHAPTER 3 OVERVIEW
DIFFERENT KINDS OF ORPHAN DRUGS
BIOLOGICAL ORPHAN DRUGS
CHEMICAL OR NON-BIOLOGICAL ORPHAN DRUGS
GLOBAL DEFINITIONS OF RARE DISEASES
U.S.
EU
TAIWAN
JAPAN
AUSTRALIA
TABLE 1 RARE DISEASES AND THEIR PREVALENCE
PERSPECTIVE AND HISTORY OF ORPHAN DRUG ACT
TABLE 2 HISTORY OF RARE DISEASES AND ORPHAN DRUG LEGISLATION
LEGISLATION IN THE U.S.
NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD)
ORPHAN DRUG ACT
TABLE 3 AMENDMENTS OF U.S. ORPHAN DRUG ACT
GLOBAL PERSPECTIVE OF ORPHAN DRUGS
EUROPE
AUSTRALIA
SINGAPORE
JAPAN
TAIWAN
CANADA
BRAZIL
SOUTH KOREA
ISRAEL
RUSSIA
KAZAKHSTAN
TABLE 4 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU,
AUSTRALIA AND JAPAN
IMPACT OF ORPHAN DRUG ACT
TABLE 5 NUMBER OF FDA ORPHAN DRUG DESIGNATIONS AND APPROVALS
PER YEAR, 1983-2014
DIFFERENCE BETWEEN ORPHAN DRUGS VS NORMAL DRUGS
TABLE 6 DIFFERENCES BETWEEN ORPHAN DRUGS AND NORMAL DRUGS
APPLICATION OF APPROVED ORPHAN DRUGS
TABLE 7 APPROVED ORPHAN DRUGS AND THEIR THERAPEUTIC USE
CHAPTER 4 REGULATORY ASPECTS
TABLE 8 MARKET APPROVALS OF ORPHAN DRUGS, 2010-2014
RECALLS
TABLE 9 RECALLS OF ORPHAN DRUGS, 2010-2014
SAFETY ALERTS
TABLE 10 SAFETY ALERTS OF ORPHAN DRUGS, 2010-2014
CHAPTER 5 NEW DEVELOPMENTS
TABLE 11 ORPHAN DRUGS IN CLINICAL TRIALS
MERGERS AND ACQUISITIONS
TABLE 12 MERGERS AND ACQUISITIONS OF ORPHAN DRUG COMPANIES, 2010-2014
CHAPTER 6 MARKET ANALYSIS
MARKET BY TYPE
MARKET OVERVIEW
MARKET REVENUE
TABLE 13 GLOBAL REVENUE FOR ORPHAN DRUGS BY TYPE, THROUGH 2019 ($ MILLIONS)
FIGURE 1 GLOBAL REVENUE FOR ORPHAN DRUGS BY TYPE, 2012-2019 ($ MILLIONS)
MARKET SHARES
TABLE 14 GLOBAL MARKET SHARE OF MAJOR ORPHAN DRUGS BY TYPE, 2013 (%)
FIGURE 2 GLOBAL MARKET SHARE OF MAJOR ORPHAN DRUGS BY TYPE, 2013 (%)
MARKET BY REGION
MARKET OVERVIEW
MARKET REVENUE
TABLE 15 GLOBAL REVENUE FOR ORPHAN DRUGS BY REGION, THROUGH 2019 ($ MILLIONS)
FIGURE 3 GLOBAL REVENUE FOR ORPHAN DRUGS BY REGION, 2012-2019 ($ MILLIONS)
MARKET SHARE
TABLE 16 GLOBAL MARKET SHARE OF ORPHAN DRUGS BY REGION, 2013 (%)
FIGURE 4 GLOBAL MARKET SHARE OF ORPHAN DRUGS BY REGION, 2013 (%)
BIOLOGICAL ORPHAN DRUGS
MARKET OVERVIEW
Ilaris (canakinumab)
Proleukin (aldesleukin)
Nplate (romiplostim)
Enbrel (etanercept)
Neupogen (filgrastim)
Sensipar/Mimpara (cinacalcet)
Neulasta (pegfilgrastim)
Xgeva (denosumab)
Epogen/Procrit (epoetin alfa)
Yervoy (ipilimumab)
Betaseron/Betaferon (interferon beta-1b)
Intron A (interferon Alfa-2b)
Kogenate (Recombinant Anti-hemophilic Factor)
Remicade (infliximab)
Rebetol (ribavirin)
Novoseven (Recombinant Coagulation Factor VIIa)
Norditropin, Saizen, Genotropin and Nutropin (Somatotropin
Recombinant Deoxyribo Nucleic Acid (rDNA))
Benefix (Recombinant Coagulation Factor IX)
Avastin (bevacizumab)
Rituxan/Mab Thera (rituximab)
Herceptin (trastuzumab)
Aldurazyme (laronidase)
Fabrazyme (agalsidase beta)
Myozyme/Lumizyme (aglucosidase alpha)
Thymoglobulin/Thymoglobulime (Anti-thymocyte Globulin)
Mozobil (plerixafor injection)
Naglazyme (galsulfase)
Soliris (eculizumab)
Humira (adalimumab)
Elaprase (idursulfase) and VPRIV (velaglucerase alfa)
Replagal (agalsidase alfa)
Gammagard Liquid (Human Immune Globulin Intravenous)
Avonex and Rebif (interferon beta 1-alpha)
Other Biological Orphan Drugs
MARKET REVENUE
TABLE 17 GLOBAL REVENUE OF BIOLOGICAL ORPHAN DRUGS BY ACTIVE INGREDIENT, THROUGH 2019 ($ MILLIONS)
MARKET SHARE
TABLE 18 GLOBAL MARKET SHARES OF BIOLOGICAL DRUGS, 2013 (%)
FIGURE 5 GLOBAL MARKET SHARES OF NON-BIOLOGICAL DRUGS, 2013 (%)
MARKET BY REGION
Market Overview
Market Revenue
TABLE 19 GLOBAL REVENUE FOR BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH 2019 ($ MILLIONS)
FIGURE 6 GLOBAL REVENUE FOR BIOLOGICAL ORPHAN DRUGS BY REGION, 2012-2019 ($ MILLIONS)
Market Share
TABLE 20 GLOBAL MARKET SHARE OF BIOLOGICAL ORPHAN DRUG MARKET BY REGION, 2013 (%)
FIGURE 7 GLOBAL MARKET SHARE OF BIOLOGICAL ORPHAN DRUG MARKET BY REGION, 2013 (%)
NON-BIOLOGICAL ORPHAN DRUGS
MARKET OVERVIEW
Exjade (deferasirox)
Sandostatin LAR (octreotide acetate)
Afinitor/Votubia (everolimus)
TOBI (tobramycin)
Zometa/Zomera/Aclasta/Reclast (zoledronic or zoledronate)
Gleevec/Glivec (imatinib mesylate)
Copaxone (glatiramer acetate)
Tasigna (nilotinib)
Sprycel (dasatanib)
Onfi (clobazam)
Revlimid (lenalidomide)
Thalomid (thalidomide)
Vidaza (azacitidine for injection)
Pomalyst/Imnovid (pomalidomide)
Flolan (epoprostenol sodium)
Volibris/Letairis/Pulmonext (ambrisentan)
Lamictal (lamatrigine)
Promacta/Revolade (eltrombopag)
Votrient (pazopanib)
Velcade (bortezomib)
Aromasin (exemestane)
Celebrex (Celecoxib)
Xalkori (crizotinib)
Cerezyme (imiglucerase for injection)
Kuvan (6R-BH4)
Tracleer (bosentan)
Opsumit (macitentan)
Zavesca (miglustat)
Ventavis (iloprost)
Kalydeco (ivacaftor)
Treanda (bendamustine), Copaxone (glatiramer)
Lialda (mesalamine)
Other Non-Biological Orphan Drugs
MARKET REVENUE
TABLE 21 GLOBAL REVENUE FOR NON-BIOLOGICAL ORPHAN DRUGS BY ACTIVE INGREDIENT, THROUGH 2019 ($ MILLIONS)
MARKET SHARE
TABLE 22 GLOBAL MARKET SHARES OF NON-BIOLOGICAL DRUGS, 2013 (%)
FIGURE 8 GLOBAL MARKET SHARES OF NON-BIOLOGICAL DRUGS, 2013 (%)
MARKET BY REGION
Market Overview
Market Revenue
TABLE 23 GLOBAL REVENUE FOR NON-BIOLOGICAL ORPHAN DRUGS BY REGION, THROUGH 2019 ($ MILLIONS)
FIGURE 9 GLOBAL REVENUE FOR NON-BIOLOGICAL ORPHAN DRUGS BY REGION, 2012-2019 ($ MILLIONS)
Market Share
TABLE 24 GLOBAL MARKET SHARE OF NON-BIOLOGICAL ORPHAN DRUG MARKET BY REGION, 2013 (%)
FIGURE 10 GLOBAL MARKET SHARE OF NON-BIOLOGICAL ORPHAN DRUG MARKET BY REGION, 2013 (%)
CHAPTER 7 INDUSTRY STRUCTURE
BIOLOGICAL ORPHAN DRUGS
MARKET LEADERS
TABLE 25 LEADING MANUFACTURERS/SUPPLIERS OF BIOLOGICAL ORPHAN DRUGS, 2013
MARKET SHARE
TABLE 26 GLOBAL MARKET SHARE OF THE MANUFACTURERS/SUPPLIERS FOR BIOLOGICAL ORPHAN DRUGS, 2013 (%)
FIGURE 11 GLOBAL MARKET SHARE OF MANUFACTURERS/SUPPLIERS FOR BIOLOGICAL ORPHAN DRUGS, 2013 (%)
NON-BIOLOGICAL ORPHAN DRUGS
MARKET LEADERS
TABLE 27 LEADING MANUFACTURERS/SUPPLIERS OF NON-BIOLOGICAL ORPHAN DRUGS, 2013
MARKET SHARE
TABLE 28 GLOBAL MARKET SHARE OF THE MANUFACTURERS/SUPPLIERS FOR NON-BIOLOGICAL ORPHAN DRUGS, 2013 (%)
FIGURE 12 MARKET SHARE OF MANUFACTURERS/SUPPLIERS FOR NON-BIOLOGICAL ORPHAN DRUGS, 2013 (%)
CHAPTER 8 MARKET BY APPLICATION
GLOBAL ORPHAN DRUGS MARKET BY APPLICATION
MARKET OVERVIEW
MARKET REVENUE
TABLE 29 GLOBAL REVENUES FOR ORPHAN DRUGS BY MEDICAL APPLICATION, THROUGH 2019 ($ MILLIONS)
FIGURE 13 GLOBAL REVENUES FOR ORPHAN DRUGS BY MEDICAL APPLICATION, 2012-2019 ($ MILLIONS)
MARKET SHARE
TABLE 30 GLOBAL MARKET SHARE OF ORPHAN DRUGS BY MEDICAL APPLICATION, 2013 (%)
FIGURE 14 GLOBAL MARKET SHARE OF ORPHAN DRUGS BY MEDICAL APPLICATION, 2013 (%)
ONCOLOGY
TABLE 31 ONCOLOGY DISEASES
Market Overview
Market Revenue
TABLE 32 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN ONCOLOGY, THROUGH 2019 ($ MILLIONS)
PULMONOLOGY
TABLE 33 PULMONOLOGY DISEASES
Market Overview
Market Revenue
TABLE 34 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN PULMONOLOGY, THROUGH 2019 ($ MILLIONS)
FIGURE 15 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN PULMONOLOGY, 2012-2019 ($ MILLIONS)
CARDIOLOGY AND NEPHROLOGY
TABLE 35 CARDIOLOGY AND NEPHROLOGY DISEASES
Market Overview
Market Revenue
TABLE 36 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN CARDIOLOGY AND NEPHROLOGY, THROUGH 2019 ($ MILLIONS)
FIGURE 16 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN CARDIOLOGY AND NEPHROLOGY, 2012-2019 ($ MILLIONS)
AUTOIMMUNE DISORDERS
TABLE 37 AUTOIMMUNE DISORDERS
Market Overview
Market Revenue
TABLE 38 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN AUTOIMMUNE DISORDERS, THROUGH 2019 ($ MILLIONS)
FIGURE 17 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN AUTOIMMUNE DISORDERS, 2012-2019 ($ MILLIONS)
BLOOD DISORDERS
TABLE 39 BLOOD DISORDERS
Market Overview
Market Revenue
TABLE 40 GLOBAL REVENUE OF ORPHAN DRUGS USED IN BLOOD DISORDERS, THROUGH 2019 ($ MILLIONS)
FIGURE 18 GLOBAL REVENUE OF ORPHAN DRUGS USED IN BLOOD DISORDERS, 2012-2019 ($ MILLIONS)
NEUROLOGY
TABLE 41 NEUROLOGICAL DISEASES
Market Overview
Market Revenue
TABLE 42 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN NEUROLOGICAL DISORDERS, THROUGH 2019 ($ MILLIONS)
FIGURE 19 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN NEUROLOGICAL DISORDERS, 2012-2019 ($ MILLIONS)
HORMONAL DISORDERS AND INFECTIONS
TABLE 43 HORMONAL DISORDERS AND INFECTIONS
Market Overview
Market Revenue
TABLE 44 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN HORMONAL DISORDERS AND INFECTIONS, THROUGH 2019 ($ MILLIONS)
FIGURE 20 GLOBAL REVENUE FOR ORPHAN DRUGS USED IN HORMONAL DISORDERS AND INFECTIONS, 2012-2019 ($MILLIONS)
CHAPTER 9 PATENT ANALYSIS
PATENTS BY YEAR
TABLE 45 NUMBER OF SIGNIFICANT U.S. PATENTS BY YEAR, 2010-2014
FIGURE 21 NUMBER OF SIGNIFICANT U.S. PATENTS BY YEAR, 2010-2014
PATENTS BY TYPE/CATEGORY
TABLE 46 NUMBER OF U.S. PATENTS BY CATEGORY, 2010-2014
FIGURE 22 NUMBER OF U.S. PATENTS BY CATEGORY, 2010-2014
PATENTS BY COMPANY
TABLE 47 NUMBER OF U.S. PATENTS BY COMPANY, 2010-2014
PATENTS BY COUNTRY
TABLE 48 NUMBER OF U.S. PATENTS BY COUNTRY, 2010-2014
FIGURE 23 NUMBER OF U.S. PATENTS BY COUNTRY, 2010-2014 ($ MILLIONS)
TABLE 49 PATENT SHARES BY COUNTRY, 2010-2014 (NO/%)
FIGURE 24 PATENT SHARES BY COUNTRY, 2010-2014 (%)
PATENTS BY ASSIGNEE
TABLE 50 NUMBER OF PATENTS BY ASSIGNEE, 2010-2014
FIGURE 25 NUMBER OF PATENTS BY ASSIGNEE, 2010-2014
CHAPTER 10 CURRENT TRENDS
FACTORS THAT ARE BOOSTING THE ORPHAN DRUG MARKET
ORPHAN DRUG ACT AND SIMILAR LEGISLATIONS
TECHNOLOGICAL ADVANCES AND GENETIC CODE
GENERIC COMPETITION
PATENT EXPIRATIONS
PREMIUM PRICING
INNOVATIONS IN MANUFACTURING TECHNOLOGIES
COLLABORATIONS AND LICENSING AGREEMENTS
MERGERS AND ACQUISITIONS
CHALLENGES
LACK OF TRAINED PROFESSIONALS
VULNERABLE TARGET GROUPS
MULTIPLE USAGES
REGULATORY CHALLENGE
CHAPTER 11 COMPANY PROFILES
ACORDA THERAPEUTICS
ACTELION PHARMACEUTICALS LTD.
AEGERION PHARMACEUTICALS
ALEXION PHARMACEUTICALS
AMGEN
BAXTER INTERNATIONAL
BAYER HEALTHCARE PHARMACEUTICALS
BIOGEN IDEC
BIOMARIN PHARMACEUTICAL INC.
BRISTOL-MYERS SQUIBB
CANGENE CORP.
CELGENE
GENZYME
GILEAD SCIENCES INC.
GLAXOSMITHKLINE
JOHNSON & JOHNSON
LUNDBECK LLC
MERCK
NOVARTIS
NOVO NORDISK INC.
ONYX THERAPEUTICS
PFIZER INC.
ROCHE
SEATTLE GENETICS
SHIRE PLC
TAKEDA PHARMACEUTICAL U.S.A. INC.
VECTURA GROUP PLC
VERTEX PHARMACEUTICALS
CHAPTER 12 ABBREVIATIONS

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