You are here

Global Orphan Drugs (Biologics, and Non-Biologics) Markets, 2016-2019 & 2024 - Select Product Approvals/Launches, Recent Industry Activity, & Focus on Key Players

DUBLIN, May 28, 2019 /PRNewswire/ -- The "Orphan Drugs - Market Analysis, Trends, and Forecasts" report has been added to's offering.

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. (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)

Key Topics Covered:

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

Regulatory Incentives
A Push in the Right Direction
Growing Unmet Medical Needs
Fulfilling the Gap
Rising Incidence 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
Pre-Symptomatic Screening to Expand Market Base
Asia's Rising Awareness and Growing Population to Drive Sales

Drug Repurposing Provides Therapies for Rare Diseases
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
Medical Advancements Foster Growth
Full Reimbursements Critical for Spurring Uptake of High- Priced Orphan Drugs
Regulators to Scrutinize Exorbitant Pricing of Ultra-Orphan 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
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

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

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

The Need for Regulation
The Orphan Drug Act of 1983
The Amended Pharmaceutical Law 1993 (Japan)
The Australian Orphan Drugs Program
The European Regulation on Orphan Medicinal Products

Orphan Drugs
A Prologue
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
Multiple Myeloma
Atypical Hemolytic Uremic Syndrome (aHUS)
Chronic Myelogenous Leukemia (CML)
Cushing's syndrome
Short Bowel Syndrome (SBS)
Duchenne's Muscular Dystrophy (DMD)
Homozygous Familial Hypercholesterolemia (hoFH)
Idiopathic Pulmonary Fibrosis (IPF)
Metastatic Melanoma
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Ultra-Orphan Drugs
Orphan Drug Categorization
Designation of Orphan Status
Personalized Medicine and Orphan Drugs
Regulatory Differences between the US and EU

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

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


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)

For more information about this report visit

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Laura Wood, Senior Manager  

For E.S.T Office Hours Call +1-917-300-0470
For U.S./CAN Toll Free Call +1-800-526-8630
For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907
Fax (outside U.S.): +353-1-481-1716

Cision View original content:

SOURCE Research and Markets