2014

Global Orphan Drugs Industry

NEW YORK, Oct. 8, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Global Orphan Drugs Industry
http://www.reportlinker.com/p01658723/Global-Orphan-Drugs-Industry.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_and_Medication

This report analyzes the worldwide markets for Orphan Drugs in US$ Million.

The report provides separate comprehensive analytics for the US, Europe, Japan, and Rest of World. Annual estimates and forecasts are provided for the period 2010 through 2018. Also, a six-year historic analysis is provided for these markets.

The report profiles 113 companies including many key and niche players such as AbbVie Inc., Actelion Pharmaceuticals Ltd., Amgen Inc., AstraZeneca plc, Bayer HealthCare AG, Biogen Idec Inc., Bristol-Myers Squibb Company, Boehringer Ingelheim GmbH, Celgene Corporation, CEL-SCI Corporation, Eli Lilly and Company, F. Hoffmann-La Roche Ltd., GlaxoSmithKline Plc, Johnson & Johnson, Merck & Co., Inc., Merck Serono International S.A., Novartis AG, Pfizer Inc., Recordati S.p.A., Orphan Europe, Sanofi SA, and Genzyme Corporation.

Market data and analytics are derived from primary and secondary research.

Company profiles are primarily based on public domain information including company URLs.

I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS
Study Reliability and Reporting Limitations I-1
Disclaimers I-2
Data Interpretation & Reporting Level I-2
Quantitative Techniques & Analytics I-3
Product Definitions and Scope of Study I-3

II. EXECUTIVE SUMMARY

1. INDUSTRY OVERVIEW II-1
Orphan Drugs - An Industry Jumpstarted by Regulations II-1
Overview of Orphan Drugs Policy Worldwide II-2
Table 1: Global Market for Orphan Drugs (2012): Percentage
Breakdown of Value Sales for Novartis, Roche, Pfizer,
Celgene, Merck KGaA, Biogen Idec, Eli Lilly, Amgen and
Others (includes corresponding Graph/Chart) II-3

Table 2: Global Market for Orphan Drugs (2004-2013):
Percentage Share of Orphan Drugs in Pharmaceutical Industry
Revenues (includes corresponding Graph/Chart) II-3
Select Future Orphan Drug Launches: 2013E-2015F II-4
Table 3: Top 5 Orphan Drug Blockbusters (2012): Ranked by
Revenue (includes corresponding Graph/Chart) II-4
Select Orphan Drug Candidates in Phase III Clinical Trials II-5
Select Orphan Drug Candidates in Phase II Clinical Trials II-5
Select Orphan Drug Candidates in Phase I Clinical Trials II-5
Table 4: Orphan Drug Designation: Percentage Breakdown by
Disease (includes corresponding Graph/Chart) II-6

2. GROWTH DRIVERS AND INHIBITORS II-7
Regulatory Incentives - A Push in the Right Direction II-7
Growing Unmet Medical Needs - Fulfilling the Gap II-7
Orphan Drug Pricing and Reimbursements - A Cut above the Rest II-7
Table 5: Select Orphan Drugs Prices (includes corresponding
Graph/Chart) II-8
Asia's Rising Awareness and Growing Population to Drive Sales II-9
Rise in Incidences of Rare forms of Cancer - Opportunities Galore II-9
Table 6: Orphan Drugs Market by Indication: 2012 (includes
corresponding Graph/Chart) II-10

Table 7: Global Cancer Incidence by Region: 2012 (includes
corresponding Graph/Chart) II-10

Table 8: Cancer Incidence in Developed Nations by Site: 2012
(includes corresponding Graph/Chart) II-11
Rise in Incidence of Renal Cell Carcinoma II-11
Unmet Needs in AML II-12
Aging Population Offers Growth Potential II-12
Table 9: Global Population Statistics for the 65+ Age Group:
2012 (in Million) (includes corresponding Graph/Chart) II-13
Pre-Symptomatic Screening to Expand Market Base II-14

3. CHALLENGES TO ORPHAN DRUG DEVELOPMENT II-15
Design & Execution Challenges Impede Clinical Trials II-15
Recruitment Challenges II-15
Regulatory Challenges II-15
High Cost of Drug Development - A Barrier for Growth II-16
Continuing Financial Crisis Could Affect Orphan Drug Prices II-16

4. CURRENT MARKET TRENDS II-17
Interest of Major Pharmaceutical Firms Signal a Shift in
Commercial Dynamics II-17
Select Acquisitions/Mergers in 2010-2011 II-17
International Collaboration - The Way Forward II-18
Treating Autism - The Next Big Thing? II-18
Sequencing Technology - The New Research Tool for Orphan Drugs II-19
Innovations in Biotechnology - A Boon for Orphan Drug Research II-19
Aging Gene Linked to Myeloma II-19
Antibody with Potential to Treat Multiple Myeloma II-19
Potential Treatment for Cystic Fibrosis Patients Infected
with Mycobacterium abscessus II-20
Genetic Variants to Improve Treatment of Non-small Cell Lung
Cancer II-20

5. BREAKTHROUGHS IN ORPHAN DISEASE TREATMENT - A RAY OF HOPE II-21
Telaprevir and Boceprevir for Hepatitis C II-21
Fingolimod for Multiple Sclerosis II-21
Dabigatran and Rivaroxaban to Prevent Strokes in Atrial
Fibrillation Patients II-21
Ipilimumab and Vemurafenib for Melanoma II-21
Sipuleucel-T for Prostate Cancer II-22
Crizotinib for Non-Small Cell Lung Cancer II-22
Select List of FDA Approved Breakthroughs (2012) II-22

6. REGULATORY SCENARIO II-23
The Need for Regulation II-23
US - The Orphan Drug Act of 1983 II-23
The Amended Pharmaceutical Law 1993 (Japan) II-24
The Australian Orphan Drugs Program - 1997 II-24
The European Regulation on Orphan Medicinal Products -1999 II-24

7. PRODUCT OVERVIEW II-25
Orphan Drugs - A Prologue II-25
Table 10: Number of Incidences Determining the Orphan Status
of a Disease by Region (includes corresponding Graph/Chart) II-25

Table 11: Select Orphan Diseases and Their Known Rate of
Prevalence (includes corresponding Graph/Chart) II-26
Select Orphan Disease Definitions II-26
Glioblastoma Multiforme II-26
Cystic Fibrosis II-26
Lyosomal Storage Disorders II-27
Fabry Disease II-27
Gaucher's Disease II-27
Pompe Disease II-27
Huntington's Disease II-27
Lymphoma II-27
Multiple Myeloma II-27
Atypical Hemolytic Uremic Syndrome (aHUS) II-28
Chronic Myelogenous Leukemia (CML) II-28
Cushing's syndrome II-28
Short Bowel Syndrome (SBS) II-28
Duchenne's Muscular Dystrophy (DMD) II-28
Hemophilia II-28
Homozygous Familial Hypercholesterolemia (hoFH) II-28

Idiopathic Pulmonary Fibrosis (IPF) II-28
Metastatic Melanoma II-29
Paroxysmal Nocturnal Hemoglobinuria (PNH) II-29
Ph+ALL II-29
Ultra-Orphan Drugs II-29
Table 12: Select Extremely Rare Diseases with Global
Registered Cases but No Available Prevalence Data (includes
corresponding Graph/Chart) II-29
Orphan Drug Categorization II-30
Select NMEs Approved for Orphan Indications in 2013 II-30
Designation of Orphan Status II-30
Personalized Medicine and Orphan Drugs II-30
Drugs Designated with Orphan Status: 2010-2012 II-31

8. RECENT ORPHAN DESIGNATIONS & APPROVALS II-35
FDA Grants Approval for Kynamro of Genzyme II-35
FDA Accepts Biogen's BLA for rFIXFc II-35
Boehringer Ingelheim Pharmaceuticals Obtains Priority Review
for afatinib II-35
Metreleptin Obtains FDA Priority Review II-35
Celgene's POMALYST® Receives FDA Approval II-36
Diffusion Pharmaceuticals Secures Orphan Drug Designation from
FDA for TSC II-36
Aerial Biopharma Obtains Orphan Designation from FDA for ARL-
N05 for Narcolepsy II-36
NPS Pharmaceuticals Secures NDA Acceptance for GATTEX® II-36
Morphotek Obtains Orphan Drug Designation for Amatuximab for
Mesothelioma II-37
DARA BioSciences Submits Orphan Drug Application for KRN5500 II-37
UCB's Keppra® Obtains Approval from US FDA II-37
Pfizer Obtains US FDA Approval for Bosulif II-37
Novartis' Signifor® Wins FDA Approval II-37

Janssen Research & Development Files NDA for bedaquiline II-38
REMICADE® Receives Approval Recommendation from GIDAC II-38
Chugai Pharmaceutical Obtains Approval for Pulmozyme
Inhalation Solution II-38
CSL Behring Obtains Orphan Drug Designation from FDA for rIX-FP II-38
Stratatech Receives FDA Orphan Drug Designation for StrataGraft® II-39
Andromeda Biotech Obtains Orphan Drug Designation for DiaPep277® II-39
REGENX BioSciences Secures Orphan Drug Designation for REGENX
Program II-39
Lexicon Pharmaceuticals Obtains Orphan Drug Designation for
LX1032 II-39
QLT Receives Orphan Drug Designation from FDA for iVisudyne II-40
Promedior Obtains Orphan Drug Designation from FDA for PRM-151 II-40
SK Biopharmaceuticals Secures FDA Orphan Drug Designation for
Carisbamate II-40
Ultragenyx Pharmaceutical Receives Orphan Drug Designation for
UX003 II-40
Cytokinetics Obtains Orphan Medicinal Product Designation for
CK-2017357 II-41
Can-Fite BioPharma Obtains Orphan Drug Status for CF102 II-41
Glenveigh Medical Receives Orphan Drug Designation II-41
Catalyst Pharmaceutical Partners Obtains Orphan Medicinal
Product Designation for CPP-115 II-41
DelMar Pharmaceuticals Obtains Orphan Drug Designation for
VAL-083 II-42
GlaxoSmithKline Secures Approval for Promacta for Treating ITP II-42
Shire PLC Receives Marketing Approval for FIRAZYR® II-42
AstraZeneca Obtains Approval for Vandetanib II-42
Roche Secures Approval for Actemra II-43

9. RECENT INDUSTRY ACTIVITY II-44
Pfizer Signs Licensing Agreement with Repligen II-44
Shire Inks Takeover Deal with Lotus Tissue Repair II-44
Shire Concludes Collaboration with Acceleron Pharma for ACE-031 II-44
Boehringer Ingelheim and PatientsLikeMe Enter into Collaboration II-44
AbbVie Inks Development License and Option Agreement with
Receptos II-45
Abbott Concludes Separation of Pharmaceuticals Business II-45
Agility Clinical Enters into Partnership with PSR Orphan Experts II-45
Opko Health Takes Over Prolor Biotech II-45
Tranzyme Pharma Inks Agreement with Ocera Therapeutics II-46
Imaxio Takes Over Trolovol® Drug from D&A Pharma II-46
Merck Expands Line of Cancer Drugs II-46
Orphan Europe Inks Agreement with ErytechPharma II-46
Recordati Signs Agreement with Lundbeck II-47
Catalyst Pharmaceutical Partners Enters into Collaboration
with BioMarin Pharmaceutical II-47
Alnylam Pharmaceuticals and Genzyme Sign Deal II-47
Medunik Canada Inks Distribution Agreement with Orphan Europe II-48
Shire Signs Research Collaboration Agreement with Fondazione
Telethon II-48
Biogen Idec Enters Agreement to Acquire Stromedix II-48
Actelion Withdraws Offer to Acquire Trophos II-48
PTC Therapeutics, Roche and SMA Foundation Ink Licensing
Agreement II-49
Zacharon Pharmaceuticals and Pfizer Enter into Research
Partnership II-49
Boehringer Ingelheim Snaps Up Biopharmaceutical Manufacturing
and Development Plant of Amgen II-49
Biogen Idec Takes Over Biogen Dompé II-49

Genzyme and ChemRar Hi-Tech Center Ink Agreement II-50
GlaxoSmithKline Inks Definitive Agreement with Amicus
Therapeutics II-50
Jennerex Enters into Partnership with Transgene II-50
SpePharm Holding Takes Over Savene® from TopoTarget II-51
Celgene Takes Over Gloucester Pharmaceuticals II-51
Pfizer and Ergonex Pharma Enter into Agreement for Terquide II-51
Swedish Orphan Biovitrum and Biogen Idec Restructure
Collaboration Agreement II-51
Kiadis Pharma and Hospira Enter into Licensing Agreement II-52
Insmed Takes Over Transave II-52
DuoCort Pharma Enters into Agreement with Recipharm II-52
Acceleron Pharma Forms Joint Venture with Shire II-53
Swedish Orphan Biovitrum Enters into Agreement with Amgen II-53
Stromedix Secures Exclusive License for Integrin avß5 Antibody II-53
NPS Pharmaceuticals Renews Licensing Agreement with GSK II-53

10. FOCUS ON SELECT PLAYERS II-54
AbbVie Inc. (US) II-54
Actelion Pharmaceuticals Ltd (Switzerland) II-54
Amgen Inc. (US) II-55
AstraZeneca plc (UK) II-55
Bayer HealthCare AG (Germany) II-56
Biogen Idec Inc (US) II-57
Bristol-Myers Squibb Company (US) II-57
Boehringer Ingelheim GmbH (Germany) II-58
Celgene Corporation (US) II-59
CEL-SCI Corporation (US) II-59
Eli Lilly and Company (US) II-60
F. Hoffmann-La Roche Ltd. (Switzerland) II-61
GlaxoSmithKline Plc (UK) II-61
Johnson & Johnson (US) II-62
Merck & Co., Inc. (US) II-63
Merck Serono International S.A. (Switzerland) II-63
Novartis AG (Switzerland) II-64
Pfizer Inc (US) II-64
Recordati S.p.A. (Italy) II-65
Orphan Europe (France) II-65
Sanofi SA (France) II-65
Genzyme Corporation (US) II-65

11. GLOBAL MARKET PERSPECTIVE II-67
Table 13: World Recent Past, Current & Future Analysis for
Orphan Drugs by Geographic Region - US, Japan, Europe and Rest
of World Markets Independently Analyzed with Annual Sales
Figures in US$ Million for the Years 2010 through 2018
(includes corresponding Graph/Chart) II-67

Table 14: World Historic Review for Orphan Drugs by Geographic
Region - US, Japan, Europe and Rest of World Markets
Independently Analyzed with Annual Sales Figures in US$
Million for the Years 2004 through 2009 (includes
corresponding Graph/Chart) II-68

Table 15: World 15-Year Perspective for Orphan Drugs by
Geographic Region - Percentage Share Breakdown of Value Sales
for US, Japan, Europe and Rest of World Markets for Years
2004, 2013 & 2018 (includes corresponding Graph/Chart) II-69
Analysis by Product Segment II-70
Table 16: World Recent Past, Current & Future Analysis for
Orphan Biologic Drugs by Region - US, Japan, Europe and Rest
of World Markets Independently Analyzed with Annual Sales
Figures in US$ Million for the Years 2010 through 2018
(includes corresponding Graph/Chart) II-70

Table 17: World Historic Review for Orphan Biologic Drugs by
Region - US, Japan, Europe and Rest of World Markets
Independently Analyzed with Annual Sales Figures in US$
Million for the Years 2004 through 2009 (includes
corresponding Graph/Chart) II-71

Table 18: World 15-Year Perspective Orphan Biologics by Region -
Percentage Breakdown of Value Sales for US, Japan, Europe
and Rest of World Markets for the Years 2004, 2013 & 2018
(includes corresponding Graph/Chart) II-72

Table 19: World Recent Past, Current & Future Analysis for
Orphan Non-Biologic Drugs by Region - US, Japan, Europe and
Rest of World Markets Independently Analyzed with Annual Sales
Figures in US$ Million for the Years 2010 through 2018
(includes corresponding Graph/Chart) II-73

Table 20: World Historic Review for Orphan Non-Biologic Drugs
by Region - US, Japan, Europe and Rest of World Markets
Independently Analyzed with Annual Sales Figures in US$
Million for the Years 2004 through 2009 (includes
corresponding Graph/Chart) II-74

Table 21: World 15-Year Perspective Orphan Non-Biologic Drugs
by Region - Percentage Breakdown of Value Sales for US, Japan,
Europe and Rest of World Markets for the 2004, 2013 & 2018
(includes corresponding Graph/Chart) II-75

III. MARKET

1. THE UNITED STATES III-1
A.Market Analysis III-1
Current and Future Analysis III-1
Overview III-1
Table 22: US Annual Orphan Drug Approvals 1983-2013
(includes corresponding Graph/Chart) III-2
US Orphan Drugs Launched in 2013 III-3
Regulatory Overview III-3
Orphan Products Grants Program III-3
US Orphan Drugs Policy - A Snapshot III-3
Humanitarian Use Device (HUD) Program III-4
Select CDRH Approved Devices for Rare Diseases (US) III-4
Medicare Reimbursement for Orphan Drugs III-4
Reimbursement for Orphan Drugs Not on Single-Indication List III-5
Recent Orphan Drug Designations & Approvals III-5

Strategic Corporate Developments III-13
Key Players III-18
B.Market Analytics III-25
Table 23: US Recent Past, Current & Future Analysis for
Orphan Drugs by Product Segment - Biologic Drugs and
Non-Biologic Drugs Markets Independently Analyzed with
Annual Sales Figures in US$ Million for Years 2010 through
2018 (includes corresponding Graph/Chart) III-25

Table 24: US Historic Review for Orphan Drugs by Product
Segment - Biologic Drugs and Non-Biologic Drugs Markets
Independently Analyzed with Annual Sales Figures in US$
Million for Years 2004 through 2009 (includes corresponding
Graph/Chart) III-26

Table 25: US 15-Year Perspective for Orphan Drugs by Product
Segment - Percentage Breakdown of Value Sales for Biologic
Drugs and Non-Biologic Drugs for the Years 2004, 2013 & 2018
(includes corresponding Graph/Chart) III-27

2. JAPAN III-28
A.Market Analysis III-28
Current and Future Analysis III-28
Regulatory Overview III-28
Designated Orphan Agents in Japan (2012) III-28
Recent Orphan Drug Approval III-29
B.Market Analytics III-29
Table 26: Japanese Recent Past, Current & Future Analysis
for Orphan Drugs by Product Segment - Biologic Drugs and
Non-Biologic Drugs Markets Independently Analyzed with
Annual Sales Figures in US$ Million for Years 2010 through
2018 (includes corresponding Graph/Chart) III-29

Table 27: Japanese Historic Review for Orphan Drugs by
Product Segment - Biologic Drugs and Non-Biologic Drugs
Markets Independently Analyzed with Annual Sales Figures in
US$ Million for Years 2004 through 2009 (includes
corresponding Graph/Chart) III-30

Table 28: Japanese 15-Year Perspective for Orphan Drugs by
Product Segment - Percentage Breakdown of Value Sales for
Biologic Drugs and Non-Biologic Drugs for the Years 2004,
2013 & 2018 (includes corresponding Graph/Chart) III-31

3. EUROPE III-32

A.Market Analysis III-32
Current and Future Analysis III-32
Overview III-32
Table 29: Number of Designated Orphan Medicinal Products in
EU 2002-2013 (includes corresponding Graph/Chart) III-32
The European Regulation on Orphan Medicinal Products - 1999 III-33
EU Orphan Drug Regulation Overview III-33
Orphan Drug Legislation in Select EU Countries III-33
EMA Approved Orphan Drugs - 2012 & 2013 III-34
EU Initiatives for Rare Disease Drug Development III-34
Select EU Funded Rare Disease Projects - Category Wise
Listings III-35
Euro-Zone Crisis and Orphan Drug Prices III-37
Recent Orphan Drug Designations III-38
Strategic Corporate Developments III-38
Key Players III-43
B.Market Analytics III-49
Table 30: European Recent Past, Current & Future Analysis
for Orphan Drugs by Product Segment - Biologic Drugs and
Non-Biologic Drugs Markets Independently Analyzed with
Annual Sales Figures in US$ Million for Years 2010 through
2018 (includes corresponding Graph/Chart) III-49

Table 31: European Historic Review for Orphan Drugs by
Product Segment - Biologic Drugs and Non-Biologic Drugs
Markets Independently Analyzed with Annual Sales Figures in
US$ Million for Years 2004 through 2009 (includes
corresponding Graph/Chart) III-50

Table 32: European 15-Year Perspective for Orphan Drugs by
Product Segment - Percentage Breakdown of Value Sales for
Biologic Drugs and Non-Biologic Drugs for the Years 2004,
2013 & 2018 (includes corresponding Graph/Chart) III-51

4. REST OF WORLD III-52
A.Market Analysis III-52
Current and Future Analysis III-52
Asia's Rising Awareness and Growing Population to Drive Sales III-52
Regulatory Scenario in Select Countries III-52
Australia III-52
China III-53
Hong Kong III-53
Singapore III-53
South Africa III-53
South Korea III-53
Taiwan III-54
Strategic Corporate Developments III-54
B.Market Analytics III-55
Table 33: Rest of World Recent Past, Current & Future
Analysis for Orphan Drugs by Product Segment - Biologic
Drugs and Non-Biologic Drugs Markets Independently Analyzed
with Annual Sales Figures in US$ Million for Years 2010
through 2018 (includes corresponding Graph/Chart) III-55

Table 34: Rest of World Historic Review for Orphan Drugs by
Product Segment - Biologic Drugs and Non-Biologic Drugs
Markets Independently Analyzed with Annual Sales Figures in
US$ Million for Years 2004 through 2009 (includes
corresponding Graph/Chart) III-56

Table 35: Rest of World 15-Year Perspective for Orphan Drugs
by Product Segment - Percentage Breakdown of Value Sales for
Biologic Drugs and Non-Biologic Drugs for the Years 2004,
2013 & 2018 (includes corresponding Graph/Chart) III-57

IV. COMPETITIVE LANDSCAPE

Total Companies Profiled: 113 (including Divisions/Subsidiaries - 121)

The United States (61)
Canada (4)
Japan (9)
Europe (42)
- France (8)
- Germany (3)
- The United Kingdom (4)
- Italy (4)
- Rest of Europe (23)
Asia-Pacific (Excluding Japan) (3)
Middle East (2)

To order this report: Global Orphan Drugs Industry
http://www.reportlinker.com/p01658723/Global-Orphan-Drugs-Industry.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_and_Medication

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