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Orphan Drugs - Global Strategic Business Report Product Image

Orphan Drugs - Global Strategic Business Report

  • ID: 2666986
  • April 2015
  • Region: Global
  • 369 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, Japan, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2014 through 2020.

Also, a seven-year historic analysis is provided for these markets. Market data and analytics are derived from primary and secondary research. Company profiles are primarily based on public domain information including company URLs.

The report profiles 142 companies including many key and niche players such as -

- AbbVie, Inc.
- Actelion Pharmaceuticals Ltd.
- Amgen, Inc.
- AstraZeneca Plc
- Bayer HealthCare AG

Note: Product cover images may vary from those shown

ORPHAN DRUGS

A GLOBAL STRATEGIC BUSINESS REPORT

CONTENTS

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
A Prelude
List of Orphan Drug Designation Criteria in Various Countries
Table 1: Select Orphan Diseases and their Prevalence (includes corresponding Graph/Chart)
The 1983 Orphan Drugs Act (ODA) Kickstarts the Industry
Orphan Drugs Legislations Worldwide
Market Potential
Market Challenges
Competition
Table 2: Global Orphan Drugs Market by Leading Player (2013): Percentage Market Share Breakdown of Value Sales for Actelion, Alexion, Amgen, Baxter, Bayer, Biogen Idec, BMS, Celgene, Lilly, GSK, J&J, Merck, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi, Shire, vertex, and Others (includes corresponding Graph/Chart)
Table 3: Top 10 Blockbuster Orphan Drugs Ranked by Revenue: 2013 (includes corresponding Graph/Chart)
Orphan Drugs Remain Strong in the Pharmaceutical Pipeline
Table 4: Global Orphan Drugs Market (2012-2018E): Percentage Share of Orphan Drug Sales in Prescription Drugs Market (includes corresponding Graph/Chart)

2. ORPHAN DRUG FDA APPROVALS
FDA-Approved Orphan Drugs (2015)
FDA-Approved Orphan Drugs (2014)
FDA-Approved Orphan Drugs (2013)
New FDA-Approved Orphan Drugs (2012)

3. GROWTH DRIVERS AND INHIBITORS
Regulatory Incentives
A Push in the Right Direction
Growing Unmet Medical Needs
Fulfilling the Gap
Asia’s Rising Awareness and Growing Population to Drive Sales
Rise in Incidences of Rare forms of Cancer
Opportunities Galore
Select Orphan Drugs for Cancer
Rise in Incidence of Renal Cell Carcinoma
Unmet Needs in AML
Aging Population Offers Growth Potential
Table 5: Global Population Statistics for the 65+ Age Group: 2013 (includes corresponding Graph/Chart)
Pre-Symptomatic Screening to Expand Market Base

4. CURRENT MARKET TRENDS
Interest of Major Pharmaceutical Firms Signal a Shift in Commercial Dynamics
Select Mergers and Acquisitions in 2014
Full Reimbursements Critical for Spurring Uptake of High- Priced Orphan Drugs
Table 6: Select Orphan Drug Prices (Per Patient per Year)
2014 (includes corresponding Graph/Chart)
Regulators to Scrutinize Exorbitant Pricing of Ultra-Orphan Drugs
Table 7: Select Ultra-Orphan Drug Prices (Per Patient per Year)
2013 (includes corresponding Graph/Chart)
Increasing Patient Engagement
The PAG Trend
Early Access Programs Gain Ground
Gene Therapy and Bioprocessing Preferred Over Chronic Therapies
Increasing M&A Activity Consolidates the Market
International Collaboration
The Way Forward
CNS Therapeutics to Gain Focus in the Orphan Drugs Market
Treating Autism
The Next Big Thing?
Strong Market Potential for Ultra-Orphan Drugs for Amyloid Light-chain Amyloidosis
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
Pirfenidone for Idiopathic Pulmonary Fibrosis (IPF)
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

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

7. PRODUCT OVERVIEW
Orphan Drugs
A Prologue
Table 8: Number of Incidences Determining the Orphan Status of a Disease by Region (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 9: Select Ultra-Orphan Diseases with Global Registered Cases but No Available Prevalence Data (includes corresponding Graph/Chart)
Orphan Drug Categorization
Designation of Orphan Status
Personalized Medicine and Orphan Drugs

8. PRODUCT APPROVALS
EC Approves Signifor® for Acromegaly
FDA Approves Isavucanazole for Invasive Candidiasis
FDA Grants Orphan Drug Designation to MabVax’s Neuroblastoma Vaccine
SYN-005 mAb by Synthetic Biologics Granted Orphan Drug Designation by FDA
Soligenix Expands Pipeline with Orphan Drug SGX301
ABT-414 by AbbVie Granted Orphan Drug Designation
FDA Grants Orphan Drug Designation to AbbVie’s HUMIRA
Ciprofloxacin DPI by Bayer HealthCare Granted Orphan Drug Status
Volasertib Granted Orphan Drug Designation for Treatment of AML
Actelion Launches VALCHLOR™ For Treatment of Rare CTCL
EC and EMA Grant Orphan Drug Status to hGH-CTP by PROLOR
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
Celgene's 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
UCB's 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
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
EC Approves CAPRELSA For Treatment of Medullary Thyroid Cancer

9. RECENT INDUSTRY ACTIVITY
Concordia to Acquire Covis Pharma
AbbVie to Acquire Pharmacyclics
Genzyme Announces Publication of Cerdelga® Trial Results
BioMarin to Acquire Prosensa Holding
AstraZeneca to Divest Myalept™ to Aegerion
Roche to Acquire InterMune
Baxter Acquires AesRx LLC
MabVax Therapeutics and Telik Enter Merger Agreement
Hyperion Acquires Andromeda Biotech
BioAlliance Pharma Enters Merger Agreement with Topotarget
TetraLogic to Acquire Shape Pharmaceuticals
Shire Completes Acquisition of ViroPharma
Jazz Pharmaceuticals to Acquire Gentium
MedImmune Acquires Spirogen
Opko Health Takes Over Prolor Biotech
Tranzyme Pharma Inks Agreement with Ocera Therapeutics
KLS Partners Enters Collaboration Agreement with Cydan
PPMD Partners with Siren Interactive
Nimbus Discovery Enters Agreement with Shire
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
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 Acquires Stromedix

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)
Boehringer Ingelheim GmbH (Germany)
Bristol-Myers Squibb Company (US)
Celgene Corp. (US)
CEL-SCI Corporation (US)
Concordia Healthcare Corp. (Canada)
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. (Germany)
Novartis AG (Switzerland)
Pfizer, Inc. (US)
Recordati S.p.A. (Italy)
Orphan Europe (France)
Sanofi SA (France)
Genzyme Corp. (US)

11. GLOBAL MARKET PERSPECTIVE
Table 10: 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 Years 2014 through 2020 (includes corresponding Graph/Chart)
Table 11: 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 Years 2007 through 2013 (includes corresponding Graph/Chart)
Table 12: World 14-Year Perspective for Orphan Drugs by Geographic Region
Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)
Analysis by Product Segment
Table 13: World Recent Past, Current & Future Analysis for Orphan Biologic Drugs by Geographic Region
US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2020 (includes corresponding Graph/Chart)
Table 14: World Historic Review for Orphan Biologic Drugs by Geographic Region
US, Japan, Europe and Rest of World Markets Independently Analyzed With Annual Sales Figures in US$ Million for Years 2007 through 2013 (includes corresponding Graph/Chart)
Table 15: World 14-Year Perspective for Orphan Biologic Drugs by Geographic Region
Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)
Table 16: World Recent Past, Current & Future Analysis for Orphan Non-Biologic Drugs by Geographic Region
US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2020 (includes corresponding Graph/Chart)
Table 17: World Historic Review for Orphan Non-Biologic Drugs by Geographic Region
US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2007 through 2013 (includes corresponding Graph/Chart)
Table 18: World 14-Year Perspective for Orphan Non-Biologic Drugs by Geographic Region
Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)

III. MARKET

1. THE UNITED STATES
A.Market Analysis
Current and Future Analysis
Table 19: Top 10 Orphan Drugs in the US by Sales: 2014 (includes corresponding Graph/Chart)
Orphan Drug Act
A Milestone Legislation
Table 20: US Annual Orphan Drug Approvals: 1983- Q1 2015 (includes corresponding Graph/Chart)
Table 21: US Orphan Drug Approvals (2002-2013): Percentage Share Breakdown by Indication Area (includes corresponding Graph/Chart)
Select Orphan Drugs Launched in 2013 & 2014
Regulatory Overview
US Orphan Drugs Policy
A Snapshot
Orphan Products Grants Program
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
Product Approvals
Strategic Corporate Developments
Select Key Players
B.Market Analytics
Table 22: The 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 2014 through 2020 (includes corresponding Graph/Chart)
Table 23: The 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 2007 through 2013 (includes corresponding Graph/Chart)
Table 24: The US 14-Year Perspective for Orphan Drugs by Product Segment
Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)

2. JAPAN
A.Market Analysis
Current and Future Analysis
Regulatory Overview
Designated Orphan Agents in Japan (2012-2014)
Recent Orphan Drug Approval
B.Market Analytics
Table 25: 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 2014 through 2020 (includes corresponding Graph/Chart)
Table 26: 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 2007 through 2013 (includes corresponding Graph/Chart)
Table 27: Japanese 14-Year Perspective for Orphan Drugs by Product Segment
Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)

3. EUROPE
A.Market Analysis
Current and Future Analysis
Table 28: Number of Medicinal Products with European Marketing Authorization (Intended for Rare Diseases) without Orphan Designation: 2002-2014 (includes corresponding Graph/Chart)
Table 29: Percentage Breakdown of Rare Disease Medicinal Products (without Orphan Designation) with European Market Authorization by Anatomical Therapeutic Chemical Category: 2014 (includes corresponding Graph/Chart)
Table 30: Percentage Breakdown of Orphan Disease Medicinal Products with European Marketing Authorization and Orphan Designation by Anatomical Therapeutic Chemical Category: 2014 (includes corresponding Graph/Chart)
Select EMA Approved Orphan Drugs
2013- Q3 2014
The European Regulation on Orphan Medicinal Products
1999
EU Orphan Drug Regulation Overview
Orphan Drug Legislation in Select EU Countries
Orphan Drugs Legislation in Select Non-EU Countries: 2014
EU Initiatives for Rare Disease Drug Development
Select EU Funded Rare Disease Projects
Category Wise Listings
Euro-Zone Crisis and Orphan Drug Prices
Product Approvals
Strategic Corporate Developments
Select Key Players
B.Market Analytics
Table 31: 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 2014 through 2020 (includes corresponding Graph/Chart)
Table 32: 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 2007 through 2013 (includes corresponding Graph/Chart)
Table 33: European 14-Year Perspective for Orphan Drugs by Product Segment
Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2007, 2015 & 2020 (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
Orphan Drugs Legislation in Select Asia-Pacific Countries: 2014
Regulatory Scenario in Select Countries
Australia
China
Hong Kong
Singapore
South Africa
South Korea
Taiwan
Strategic Corporate Developments
B.Market Analytics
Table 34: 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 2014 through 2020 (includes corresponding Graph/Chart)
Table 35: 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 2007 through 2013 (includes corresponding Graph/Chart)
Table 36: Rest of World 14-Year Perspective for Orphan Drugs by Product Segment
Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2007, 2015 & 2020 (includes corresponding Graph/Chart)

IV. COMPETITIVE LANDSCAPE

Total Companies Profiled: 142 (including Divisions/Subsidiaries - 154)

The United States (82)
Canada (8)
Japan (9)
Europe (49)
- France (12)
- Germany (4)
- The United Kingdom (4)
- Italy (4)
- Rest of Europe (25)
Asia-Pacific (Excluding Japan) (6)

Note: Product cover images may vary from those shown
Note: Product cover images may vary from those shown

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