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Global Orphan Drugs (Biologics, and Non-Biologics) Market Analysis, Trends, and Forecasts Report 2019 - ResearchAndMarkets.com

May 27, 2019

DUBLIN--(BUSINESS WIRE)--May 27, 2019--

The “Orphan Drugs - Market Analysis, Trends, and Forecasts” report has been added to ResearchAndMarkets.com’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 https://www.researchandmarkets.com/r/hsv274

View source version on businesswire.com:https://www.businesswire.com/news/home/20190527005188/en/

CONTACT: ResearchAndMarkets.com

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Related Topics:Drug Discovery



SOURCE: Research and Markets

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