Orphan Drugs: Market Research Report

January 2019 | 436 pages | ID: O139EB269E5EN
Global Industry Analysts, Inc

Abstracts

This report analyzes the worldwide markets for Orphan Drugs in US$ Million by the following Product Segments: Biologics, and Non-Biologics.

The report provides separate comprehensive analytics for the US, Japan, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2016 through 2024.

Also, a five-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 140 companies including many key and niche players such as -

• AbbVie, Inc.
• Actelion Pharmaceuticals Ltd.
• Alexion Pharmaceuticals, Inc.
• Amgen, Inc.
• AstraZeneca Plc
• Bayer AG

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

Table 1. List of Orphan Drug Designation Criteria in Various Countries

Table 2. Select Orphan Diseases and their Prevalence (includes corresponding Graph/Chart)

Increasing Adoption of Orphan Drugs by Manufacturers
The 1983 Orphan Drugs Act (ODA) Kickstarts the Industry
Major Orphan Drugs Legislations Worldwide
Orphan Drugs Market on a Rapid Growth Trajectory

Table 3. Orphan Drug Designations: 2010 to 2016 (includes corresponding Graph/Chart)

Select Major Organizations Dealing with Rare Diseases in the US, Canada, and EU
Incentives for Orphan Drug Designation Products in the US, EU, and Japan
Market Challenges
Obstacles for Conducting Orphan Drug Research
Competition

Table 4. Global Orphan Drugs Market by Leading Brand: Percentage Market Share for 2016 (includes corresponding Graph/Chart)

Orphan Drugs Remain Strong in the Pharmaceutical Pipeline
Important Orphan Drugs in Pipeline

2. GROWTH DRIVERS

Regulatory Incentives – A Push in the Right Direction
Growing Unmet Medical Needs – Fulfilling the Gap
Rising Incidence of Rare Forms of Cancer – Opportunities Galore

Table 5. Growing Burden of Cancer Worldwide - Number of New Cancer Cases and Cancer-Related Deaths for the Year 2012 and 2030 (includes corresponding Graph/Chart)

Table 6. Common Cause of Cancer Deaths Worldwide - Number of Deaths due to Select Types of Cancer for 2015 (includes corresponding Graph/Chart)

Table 7. New Cancer Cases in the World by Affected Site: 2012 (in Thousands) (includes corresponding Graph/Chart)

Select Orphan Drugs for Cancer
Rise in Incidence of Renal Cell Carcinoma
Unmet Needs in AML
Aging Population Offers Growth Potential

Table 8. Global Population Statistics for the 65+ Age Group (2016) (includes corresponding Graph/Chart)

Pre-Symptomatic Screening to Expand Market Base
Asia’s Rising Awareness and Growing Population to Drive Sales

3. SELECT MARKET TRENDS

Drug Repurposing Provides Therapies for Rare Diseases

Table 9. Major Blockbuster Pharma Drugs Having Orphan Designation (includes corresponding Graph/Chart)

The Trend of Old Drugs Re-Surfacing as Orphan Drugs Surges
Interest of Major Pharmaceutical Firms Signal a Shift in Commercial Dynamics
Mergers and Acquisitions Drive Market Growth
Select Mergers & Acquisitions for Orphan Drugs as of the Year 2016
Growing Scrutiny over Skyrocketing Prices
Orphan Drugs with High Prices: 2016

Table 10. Few Orphan Drug Novel Therapies with Exorbitant Prices (includes corresponding Graph/Chart)

Medical Advancements Foster Growth
Full Reimbursements Critical for Spurring Uptake of High-Priced Orphan Drugs

Table 11. Orphan Drug Prices (includes corresponding Graph/Chart)

Table 12. Orphan Drugs Vs Non-Orphan Drugs Average Cost (in Dollars) per Patient per Year 2012-2016 (includes corresponding Graph/Chart)

Table 13. Orphan Drugs Vs Non-Orphan Drugs Median Cost (in Dollars) per Patient per Year 2012-2016 (includes corresponding Graph/Chart)

Regulators to Scrutinize Exorbitant Pricing of Ultra-Orphan Drugs

Table 14. US FDA Approval of Ultra-Orphan Non-Cancer Drugs

Strong Market Potential for Ultra-Orphan Drugs for Amyloid Light-chain Amyloidosis
Increasing Patient Engagement – The PAG Trend
Gene Therapy and Bioprocessing Preferred over Chronic Therapies
Companies Developing Gene Therapies
International Collaboration – The Way Forward
CNS Therapeutics to Gain Focus in the Orphan Drugs Market

Table 15. CNS Orphan Drug Designations by Therapeutic Area: 2016 (includes corresponding Graph/Chart)

Manufacturers Target Orphan Indications to Curtail Competition in Broader Indications
Manufacturers Use Orphan Disease to Target Larger Therapeutic Indications
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

4. ORPHAN DRUG FDA APPROVALS

FDA Approved Orphan Drugs (as of July 2017)
FDA Approved Orphan Drugs (2016)
FDA Approved Orphan Drugs (2015)
FDA-Approved Orphan Drugs (2015)

5. ORPHAN DRUG DESIGNATIONS

Select Orphan drug designation: 2017 (total 433 as of november 17, 2017)
Select Orphan drug designations: 2016 (total 333)

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 16. Number of Incidences Determining the Orphan Status of a Disease by Region (includes corresponding Graph/Chart)

Table 17. Definitions of Orphan Diseases in Different Countries

Orphan Drugs Research Progress
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 18. 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
Regulatory Differences between the US and EU

8. SELECT PRODUCT APPROVALS/LAUNCHES

TiGenix Receives FDA Orphan Drug Designation for Cx601
FDA Grants Orphan Drug Designation for Emerald Health Pharmaceuticals lead molecule, EHP-101, to Treat Systemic Scleroderma
RedHill Biopharma Receives FDA Orphan Drug Designation for MESUPRON
FDA Awards Orphan Drug Status to uniQure’s Potential Huntington’s Therapy
Boston Biomedical Receives Orphan Drug Designation from FDA for DSP-7888
Amicus Receives FDA Orphan Drug Status for ATB200/AT2221
Roche’s Actemra/RoActemra (tocilizumab) Receives FDA Approval to Treat CAR T Cell-induced Cytokine Release Syndrome
AGTC Receives FDA Orphan Drug Designation for Gene Therapy Product Candidate
Polyneuron’ PN-1007 Receives Orphan Drug Status from European Medicines Agency
FDA Grants Orphan Drug Designation to Versantis VS-01
Innovate Biopharmaceuticals Applies for FDA’s Orphan Drug Designation for INN-108
FDA Grants Orphan Drug Status for Biohaven’s BHV-5000
Mithra Receives Orphan Drug Designation for E4
AstraZeneca Receives Orphan Designation from EMA for inebilizumab
Boehringer Ingelheim gets FDA Orphan Drug Status for BI 836858
Biostage gets FDA Orphan Drug Designation for Cellspan Esophageal Implant
AbbVie Obtains FDA Orphan Drug Designation for Risankizumab
AbbVie gets FDA Orphan Drug Designation for Veliparib
TiGenix Receives Orphan Drug Designation for Cx601
Boehringer Ingelheim gets FDA Orphan Drug Designation for nintedanib
AstraZeneca Receives FDA Orphan Drug Designation for Selumetinib
Amarantus gets FDA Orphan Drug Designation for Eltoprazine
AM-Pharma Secures FDA & EMA Orphan Drug Designation
AstraZeneca’s Tremelimumab Receives FDA Orphan Drug Designation
BioArbitrage chooses to focus on the Dynamic Rare Disease Sector
Acer Therapeutics ACER-002 Receives FDA Orphan Drug Designation

9. RECENT INDUSTRY ACTIVITY

Ergomed Plc Plans to Acquire PSR Group BV
UDG Healthcare Acquires Cambridge BioMarketing
Horizon Pharma Acquires River Vision Development Corp. and Teprotumumab
Sun Pharma’s Indirect arm Taro Announces Acquisition of Thallion Pharmaceuticals
Johnson & Johnson Plans to Acquire Actelion
Escend Offers Opportunity to Acquire or License ES-3000
Horizon Pharma Agrees to Take Over Raptor Pharmaceutical
Savara Acquires Serendex Pharmaceuticals
InSite Vision Inks definitive Agreement with QLT Inc
Roche Plans to Take over Trophos

10. FOCUS ON SELECT PLAYERS

AbbVie, Inc. (USA)
Actelion Pharmaceuticals Ltd. (Switzerland)
Alexion Pharmaceuticals, Inc. (USA)
Amgen, Inc. (USA)
AstraZeneca Plc. (UK)
Bayer AG (Germany)
Biogen Inc. (USA)
BioMarin Pharmaceutical Inc. (USA)
Boehringer Ingelheim GmbH (Germany)
Bristol-Myers Squibb Company (USA)
Celgene Corp. (USA)
CEL-SCI Corporation (USA)
Concordia International Corp. (Canada)
Eli Lilly and Company (USA)
F. Hoffmann-La Roche Ltd. (Switzerland)
Johnson & Johnson (USA)
Merck & Co., Inc. (USA)
Merck Serono International S. A. (Germany)
Novartis AG (Switzerland)
Pfizer, Inc. (USA)
Recordati S. p. A. (Italy)
Orphan Europe (France)
Shire Plc. (Ireland)
Sanofi SA (France)
Genzyme Corp. (USA)
Vertex Pharmaceuticals (USA)

11. GLOBAL MARKET PERSPECTIVE

Table 19. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 20. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 21. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

Analysis by Product Segment

Table 22. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 23. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 24. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

Table 25. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 26. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 27. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

III. MARKET

1. THE UNITED STATES

A. Market Analysis
Current and Future Analysis

Table 28. Prevalence and Prices of Select Orphan Drugs in the US (includes corresponding Graph/Chart)

Table 29. Leading Drugs in the US Orphan Drugs Market: Market Share of Sales in Percentage for the year 2016 (includes corresponding Graph/Chart)

Orphan Drug Act – A Milestone Legislation
US Leads the Orphan Drug Approval Landscape

Table 30. Number of Orphan Drug Approvals and Designations: 1983 to 2016 (includes corresponding Graph/Chart)

Table 31. Number of FDA Approved Orphan Drugs by Type: 2013-2016 (includes corresponding Graph/Chart)

Regulatory Overview
US Orphan Drugs Policy - A Snapshot
Orphan Products Grants Program
Humanitarian Use Device (HUD) Program
Select CDRH Approved Devices for Rare Diseases
Medicare Reimbursement for Orphan Drugs
Reimbursement for Orphan Drugs Not on Single-Indication List
Orphan Drug Modernization Plan
Priority Review of Orphan Drugs Becomes Popular
FDA Review Time Reduced for Priority Review Designation

Table 32. Majority of Orphan Drugs Undergo Priority Review: 2015 (includes corresponding Graph/Chart)

Product Approvals
Strategic Corporate Developments
Select Key Players
B. Market Analytics

Table 33. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 34. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 35. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

2. JAPAN

A. Market Analysis
Current and Future Analysis
Regulatory Overview

Table 36. Comparison of Japanese Orphan Drug Designation System with the US and Europe

New Orphan Drugs in Japan: 2015-2017

Table 37. Japanese Orphan Drugs Designation by Therapeutic Area: 2016 (includes corresponding Graph/Chart)

B. Market Analytics

Table 38. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 39. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 40. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

3. EUROPE

A. Market Analysis
Current and Future Analysis

Table 41. Leading Drugs in the European Orphan Drugs Market: Market Share of Sales in Percentage for the year 2016 (includes corresponding Graph/Chart)

Table 42. European Orphan Medicinal Product Designations since 2008 (includes corresponding Graph/Chart)

Table 43. EU Orphan Designation by Therapeutic Indications: As of 2015 (includes corresponding Graph/Chart)

Orphan Drug Marketing Authorizations in Europe: As of Sept 2017
Orphan Drug Marketing Authorizations in Europe: 2016
The European Regulation on Orphan Medicinal Products - 1999
EU Orphan Drug Regulation Overview
Orphan Drugs Policies Summary in Select European Countries
Product Approvals
Strategic Corporate Developments
Select Key Players
B. Market Analytics

Table 44. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 45. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 46. 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 2011, 2017 & 2024 (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
Orphan Drugs Market Set to Grow in China
The Chinese Food and Drug Administration (CFDA)
India
Lack of Awareness Limits the Growth of Orphan Drug Development
Malaysia
Malaysia to Develop a National Framework for the Management of Rare Diseases
Thailand
Singapore
South Africa
South Korea
Taiwan
Strategic Corporate Development
Select Key Player
B. Market Analytics

Table 47. 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 2016 through 2024 (includes corresponding Graph/Chart)

Table 48. 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 2011 through 2015 (includes corresponding Graph/Chart)

Table 49. 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 2011, 2017 & 2024 (includes corresponding Graph/Chart)

IV. COMPETITIVE LANDSCAPE

Total Companies Profiled: 140 (including Divisions/Subsidiaries - 151)
The United States (77)
Canada (8)
Japan (8)
Europe (51)
  France (10)
  Germany (4)
  The United Kingdom (7)
  Italy (3)
  Rest of Europe (27)
Asia-Pacific (Excluding Japan) (3)
Middle East (4)