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Orphan Drugs - Global Strategic Business Report Product Image

Orphan Drugs - Global Strategic Business Report

  • ID: 2666986
  • August 2013
  • Region: Global
  • 252 Pages
  • Global Industry Analysts, Inc

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
- 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.

Please note: Reports are sold as single-site single-user licenses. Electronic versions require 24-48 hours as each copy is customized to the client with digital controls and custom watermarks.

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

II. Executive Summary

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

2. GROWTH DRIVERS AND INHIBITORS
Regulatory Incentives - A Push in the Right Direction
Growing Unmet Medical Needs - Fulfilling the Gap
Orphan Drug Pricing and Reimbursements - A Cut above the Rest
Table 5: Select Orphan Drugs Prices (includes corresponding Graph/Chart)
Asia's Rising Awareness and Growing Population to Drive Sales
Rise in Incidences of Rare forms of Cancer - Opportunities Galore
Table 6: Orphan Drugs Market by Indication: 2012 (includes corresponding Graph/Chart)
Table 7: Global Cancer Incidence by Region: 2012 (includes corresponding Graph/Chart)
Table 8: Cancer Incidence in Developed Nations by Site: 2012 (includes corresponding Graph/Chart)
Rise in Incidence of Renal Cell Carcinoma
Unmet Needs in AML
Aging Population Offers Growth Potential
Table 9: Global Population Statistics for the 65+ Age Group: 2012 (in Million) (includes corresponding Graph/Chart)
Pre-Symptomatic Screening to Expand Market Base

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

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

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

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

7. PRODUCT OVERVIEW
Orphan Drugs - A Prologue
Table 10: Number of Incidences Determining the Orphan Status of a Disease by Region (includes corresponding Graph/Chart)
Table 11: Select Orphan Diseases and Their Known Rate of Prevalence (includes corresponding Graph/Chart)
Select Orphan Disease Definitions
Glioblastoma Multiforme
Cystic Fibrosis
Lyosomal Storage Disorders
Fabry Disease
Gaucher's Disease
Pompe Disease
Huntington's Disease
Lymphoma
Multiple Myeloma
Atypical Hemolytic Uremic Syndrome (aHUS)
Chronic Myelogenous Leukemia (CML)
Cushing's syndrome
Short Bowel Syndrome (SBS)
Duchenne's Muscular Dystrophy (DMD)
Hemophilia
Homozygous Familial Hypercholesterolemia (hoFH)
Idiopathic Pulmonary Fibrosis (IPF)
Metastatic Melanoma
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Ph+ALL
Ultra-Orphan Drugs
Table 12: Select Extremely Rare Diseases with Global Registered Cases but No Available Prevalence Data (includes corresponding Graph/Chart)
Orphan Drug Categorization
Select NMEs Approved for Orphan Indications in 2013
Designation of Orphan Status
Personalized Medicine and Orphan Drugs
Drugs Designated with Orphan Status: 2010-2012

8. RECENT ORPHAN DESIGNATIONS & APPROVALS
FDA Grants Approval for Kynamro of Genzyme
FDA Accepts Biogen's BLA for rFIXFc
Boehringer Ingelheim Pharmaceuticals Obtains Priority Review for afatinib
Metreleptin Obtains FDA Priority Review
Celgenes POMALYST® Receives FDA Approval
Diffusion Pharmaceuticals Secures Orphan Drug Designation from FDA for TSC
Aerial Biopharma Obtains Orphan Designation from FDA for ARL
N05 for Narcolepsy
NPS Pharmaceuticals Secures NDA Acceptance for GATTEX®
Morphotek Obtains Orphan Drug Designation for Amatuximab for Mesothelioma
DARA BioSciences Submits Orphan Drug Application for KRN5500
UCBs Keppra® Obtains Approval from US FDA
Pfizer Obtains US FDA Approval for Bosulif
Novartis' Signifor® Wins FDA Approval
Janssen Research & Development Files NDA for bedaquiline
REMICADE® Receives Approval Recommendation from GIDAC
Chugai Pharmaceutical Obtains Approval for Pulmozyme Inhalation Solution
CSL Behring Obtains Orphan Drug Designation from FDA for rIX-FP
Stratatech Receives FDA Orphan Drug Designation for StrataGraft®
Andromeda Biotech Obtains Orphan Drug Designation for DiaPep277®
REGENX BioSciences Secures Orphan Drug Designation for REGENX Program
Lexicon Pharmaceuticals Obtains Orphan Drug Designation for LX1032
QLT Receives Orphan Drug Designation from FDA for iVisudyne
Promedior Obtains Orphan Drug Designation from FDA for PRM-151
SK Biopharmaceuticals Secures FDA Orphan Drug Designation for Carisbamate
Ultragenyx Pharmaceutical Receives Orphan Drug Designation for UX003
Cytokinetics Obtains Orphan Medicinal Product Designation for CK-2017357
Can-Fite BioPharma Obtains Orphan Drug Status for CF102
Glenveigh Medical Receives Orphan Drug Designation
Catalyst Pharmaceutical Partners Obtains Orphan Medicinal Product Designation for CPP-115
DelMar Pharmaceuticals Obtains Orphan Drug Designation for VAL-083
GlaxoSmithKline Secures Approval for Promacta for Treating ITP
Shire PLC Receives Marketing Approval for FIRAZYR®
AstraZeneca Obtains Approval for Vandetanib
Roche Secures Approval for Actemra

9. RECENT INDUSTRY ACTIVITY
Pfizer Signs Licensing Agreement with Repligen
Shire Inks Takeover Deal with Lotus Tissue Repair
Shire Concludes Collaboration with Acceleron Pharma for ACE-031
Boehringer Ingelheim and PatientsLikeMe Enter into Collaboration
AbbVie Inks Development License and Option Agreement with Receptos
Abbott Concludes Separation of Pharmaceuticals Business
Agility Clinical Enters into Partnership with PSR Orphan Experts
Opko Health Takes Over Prolor Biotech
Tranzyme Pharma Inks Agreement with Ocera Therapeutics
Imaxio Takes Over Trolovol® Drug from D&A Pharma
Merck Expands Line of Cancer Drugs
Orphan Europe Inks Agreement with ErytechPharma
Recordati Signs Agreement with Lundbeck
Catalyst Pharmaceutical Partners Enters into Collaboration with BioMarin Pharmaceutical
Alnylam Pharmaceuticals and Genzyme Sign Deal
Medunik Canada Inks Distribution Agreement with Orphan Europe
Shire Signs Research Collaboration Agreement with Fondazione Telethon
Biogen Idec Enters Agreement to Acquire Stromedix
Actelion Withdraws Offer to Acquire Trophos
PTC Therapeutics, Roche and SMA Foundation Ink Licensing Agreement
Zacharon Pharmaceuticals and Pfizer Enter into Research Partnership
Boehringer Ingelheim Snaps Up Biopharmaceutical Manufacturing and Development Plant of Amgen
Biogen Idec Takes Over Biogen Dompé
Genzyme and ChemRar Hi-Tech Center Ink Agreement
GlaxoSmithKline Inks Definitive Agreement with Amicus Therapeutics
Jennerex Enters into Partnership with Transgene
SpePharm Holding Takes Over Savene® from TopoTarget
Celgene Takes Over Gloucester Pharmaceuticals
Pfizer and Ergonex Pharma Enter into Agreement for Terquide
Swedish Orphan Biovitrum and Biogen Idec Restructure Collaboration Agreement
Kiadis Pharma and Hospira Enter into Licensing Agreement
Insmed Takes Over Transave
DuoCort Pharma Enters into Agreement with Recipharm
Acceleron Pharma Forms Joint Venture with Shire
Swedish Orphan Biovitrum Enters into Agreement with Amgen
Stromedix Secures Exclusive License for Integrin avß5 Antibody
NPS Pharmaceuticals Renews Licensing Agreement with GSK

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

11. GLOBAL MARKET PERSPECTIVE
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)
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)
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)
Analysis by Product Segment
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)
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)
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)
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)
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)
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)

III. MARKET

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

2. JAPAN
A.Market Analysis
Current and Future Analysis
Regulatory Overview
Designated Orphan Agents in Japan (2012)
Recent Orphan Drug Approval
B.Market Analytics
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)
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)
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)

3. EUROPE
A.Market Analysis
Current and Future Analysis
Overview
Table 29: Number of Designated Orphan Medicinal Products in EU 2002-2013 (includes corresponding Graph/Chart)
The European Regulation on Orphan Medicinal Products - 1999
EU Orphan Drug Regulation Overview
Orphan Drug Legislation in Select EU Countries
EMA Approved Orphan Drugs - 2012 & 2013
EU Initiatives for Rare Disease Drug Development
Select EU Funded Rare Disease Projects - Category Wise Listings
Euro-Zone Crisis and Orphan Drug Prices
Recent Orphan Drug Designations
Strategic Corporate Developments
Key Players
B.Market Analytics
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)
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)
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)

4. REST OF WORLD
A.Market Analysis
Current and Future Analysis
Asia's Rising Awareness and Growing Population to Drive Sales
Regulatory Scenario in Select Countries
Australia
China
Hong Kong
Singapore
South Africa
South Korea
Taiwan
Strategic Corporate Developments
B.Market Analytics
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)
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)
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)

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)

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