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Market Research Report

Orphan Drug Market Analysis and Forecast to 2025

Published by Kelly Scientific Publications Product code 979793
Published Content info 197 Pages
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Orphan Drug Market Analysis and Forecast to 2025
Published: December 1, 2020 Content info: 197 Pages
Description

This industry analysis report tackles the major orphan drugs within the rare disease therapeutic market and encompasses the most prevalent and dominant therapies in this space. The market value for the current major orphan therapeutic space is analysed and forecast to 2025 with corresponding CAGR percentages. Market details are provided by drug type, therapeutic area, geography and also specific sales and forecast of the major drugs in the field. Key industry players and developments are covered along with the growing pipeline and clinical trials. Regional insights and approval trends are given, as are the strengths, opportunities and challenges of this growing market.

The scope of the report provides in-depth analysis of the therapeutics space by value and forecast to 2025 that is sub divided into:

  • Small molecules
  • Antibodies
  • Oligonucleotides
  • Gene therapy
  • Cell therapy

The report reviews the most dominant therapeutics in each sub-market and provides clinical evaluation, sales forecast and market share to 2025. It gives a comparison between the current market values of the main therapies and how the landscape will change by 2025 with the launch of new pipeline agents. This includes some of the main blockbusters such as

  • Revlimid
  • Trikafta
  • Darzalex
  • Spinraza
  • Hemlibra
  • Venclexta
  • Soliris
  • Adcetris
  • Jakafi
  • Lenvima
  • Lynparza
  • Ultomiris
  • Yescarta
  • Pomalyst
  • Vyndaqel
  • Ninlaro
  • Liso-cel

Therefore the reader is provided with data on the current main dominant therapeutics in the area and also the drugs that will dominate the space by 2025. This analysis is provided for each sub-therapeutic area. Geographical breakdown analysis is further provided and is segmented into North America, Europe, Asia Pacific and the Rest of the World.

The scope of the report also includes the orphan drug market by indication and disease type with specific detail given to oncology, hematological disorders, CNS, respiratory, immunomodulation, cardiovascular, musculoskeletal, anti-infectives, endocrine and gastrointestinal disease.

Key players and company profiles in the rare disease market are provided including:

  • Bristol Myers Squibb
  • Roche/Genentech
  • Alexion
  • Janssen J&J
  • Biogen
  • Novartis
  • AstraZeneca Merck
  • Eisai
  • Takeda
  • Abbvie Genentech
  • Gilead (Kite)
  • Pfizer
  • Vertex
  • Argenx

The report also includes a detailed description of the following trends and market shapers:

  • USA (FDA) and European (EMA) Orphan Drug Approvals
  • Venture Funding Increasing Over Time
  • Therapeutic Areas Dominating Approval
  • Orphan Drug Designation Trends
  • Repurposing Existing Therapeutics
  • Japan Approval Trends
  • South Korea Approval Trends
  • China Approval Trends

The growing rare disease pipeline and clinical trial environment is evaluated with specific consideration given to:

  • Small Molecules and Multiple Myeloma
  • Gene and Cell Therapy in Melanoma
  • Gene and Cell Therapy in ADA-SCID
  • Gene and Cell Therapy agents in Hemophilia A and B
  • CAR-T Therapy agents in Pancreatic Cancer
  • CAR-T Therapy agents in Multiple Myeloma
  • Gene and Cell Therapies in Duchenne Muscular Dystrophy
  • RPE65 Genetic Retinal Mutation
  • Rare Eye Disorders
  • Spinal Muscular Atrophy
  • Sickle Cell Anaemia
  • Cystic Fibrosis
  • Erythropoietic Protoporphyria
  • Guillain-Barre Syndrome
  • Scleroderma
  • Alpha-1 Antitrypsin Deficiency
  • Juvenile Idiopathic Arthritis
Table of Contents

Table of Contents

1.0 Executive Summary

2.1 USA (FDA) and European (EMA) Orphan Drug Approvals

  • 2.2 Venture Funding Increasing Over Time
  • 2.3 Therapeutic Areas Dominating Approval
  • 2.4 Orphan Drug Designation Trends
  • 2.5 Repurposing Existing Therapeutics
  • 2.6 Japan Approval Trends
  • 2.7 South Korea Approval Trends
  • 2.8 China Approval Trends

3.0 Rare Disease Market Analysis

  • 3.1 Major Orphan Drug Market to 2025
  • 3.2 Orphan Small Molecule Market Analysis to 2025
  • 3.3 Orphan Antibody Market Analysis to 2025
  • 3.4 Orphan Gene Therapy Market Analysis to 2025
  • 3.5 Orphan Cell Therapy Market Analysis to 2025
  • 3.6 Orphan Oligonucleotide Therapy Market Analysis to 2025
  • 3.7 Geographical Breakdown of Rare Disease Market to 2025
  • 3.8 Orphan Drug Market by Indication and Disease Type
  • 3.9 Key Market Players
    • 3.9.1 Bristol-Myers Squibb
    • 3.9.2 Roche/Genentech
    • 3.9.3 Alexion
    • 3.9.4 J&J/Janssen Biotech
    • 3.9.5 Biogen
    • 3.9.6 Novartis
    • 3.9.7 AstraZeneca
    • 3.9.8 Eisai
    • 3.9.9 Takeda
    • 3.9.10 Abbvie
    • 3.9.11 Gilead
    • 3.9.12 Pfizer
    • 3.9.13 Vertex Pharmaceuticals
    • 3.9.14 Argenix

4.0 Pipeline Agents in Clinical Trials by Condition, Intervention and Phase

  • 4.1 Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
  • 4.2 Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
  • 4.3 Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
  • 4.4 Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
  • 4.5 Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
  • 4.6 Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
  • 4.7 Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
  • 4.8 Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A and B
  • 4.9 Select Phase III Clinical Trials in Cystic Fibrosis
  • 4.10 Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
  • 4.11 Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
  • 4.12 Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
  • 4.13 Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular Dystrophy
  • 4.14 Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
  • 4.15 Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
  • 4.16 Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
  • 4.17 Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
  • 4.18 Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin Deficiency
  • 4.19 Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic Arthritis

5.0 Pipeline Therapeutics Analysis

6.0 Future Outlook

List of Exhibits

  • Exhibit 2.1: Number of FDA and EMA Orphan Drug Approvals from 2001-2018
  • Exhibit 2.2: FDA Priority, Accelerated, Breakthrough and Fast Track Regulatory Reviews for Rare Disease from 2008-2017
  • Exhibit 2.3: EMA PRIME, Accelerated Assessment, Exceptional Circumstance or Conditional Marketing Approval Reviews for Rare Disease from 2000-2018
  • Exhibit 2.4: Venture Funding into Rare Disorders, Excluding Oncology, 2013-2018
  • Exhibit 2.5: FDA Orphan Drug Approvals by Therapeutic Area
  • Exhibit 2.6: EMA Orphan Drug Approvals by Therapeutic Area
  • Exhibit 2.7: Number of FDA Approved Rare Disease Designations by Drug Type 2017-2020
  • Exhibit 2.8: Number of FDA Applications for Orphan Designation 2020
  • Exhibit 2.9: Number of Orphan FDA Designated Agents 20203
  • Exhibit 2.10: Percentage of FDA Applications for Orphan Designation 2020
  • Exhibit 2.11: FDA Approved Agents for Rare Disease Indications 2020
  • Exhibit 2.12: FDA Approved Agents for Rare Disease Indications 2019
  • Exhibit 2.13: FDA Approved Agents for Rare Disease Indications 2018
  • Exhibit 2.14: FDA Approved Agents for Rare Disease Indications 2017
  • Exhibit 2.15: EMA Approved Rare Disease Therapies to Date
  • Exhibit 3.1: Major Rare Disease Therapeutic Market by Drug Class 2018 - 2025
  • Exhibit 3.2 Major Rare Disease Therapeutic Market by Small Molecules, Antibodies, Gene Therapies, Cell Therapies and Oligonucleotides, 2018-2025
  • Exhibit 3.3: Percentage Market Share of Rare Disease Drugs by Subtype, 2018 Vs 2025
  • Exhibit 3.4: Top Rare Disease Drug Sales ($ Billion) 2018
  • Exhibit 3.5: Top Rare Disease Drug Sales Forecast ($ Billion) 2025
  • Exhibit 3.6: Current Small Molecules Dominating the Rare Disease Market and Forecast to 2025
  • Exhibit 3.7: Future Small Molecules within the Rare Disease Market and Forecast to 2025
  • Exhibit 3.8: Small Molecule Orphan Drug Market 2018-2025
  • Exhibit 3.9: Current & Future Antibodies Dominating the Rare Disease Market and Forecast to 2025
  • Exhibit 3.10: Orphan Antibody Market Analysis 2018-2025
  • Exhibit 3.11: Current & Future Gene Therapies Dominating the Rare Disease Market and Forecast to 2025
  • Exhibit 3.12: Orphan Gene Therapy Market Analysis 2018-2025
  • Exhibit 3.13: Current & Future CART Cell Therapies Dominating the Rare Disease Market and Forecast to 2025
  • Exhibit 3.14: Orphan Cell Therapy Market Analysis 2018-2025
  • Exhibit 3.15: Current & Future Oligonucleotide Therapies Dominating the Rare Disease Market and Forecast to 2025
  • Exhibit 3.16 Orphan Oligonucleotide Therapy Market Analysis 2018-2025
  • Exhibit 4.1: Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
  • Exhibit 4.2: Select Phase III Clinical Trials Investigating Small Antibodies and Multiple Myeloma
  • Exhibit 4.3: Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
  • Exhibit 4.4: Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
  • Exhibit 4.5: Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
  • Exhibit 4.6: Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
  • Exhibit 4.7: Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
  • Exhibit 4.8: Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
  • Exhibit 4.9: Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A/B
  • Exhibit 4.10: Select Phase III Clinical Trials in Cystic Fibrosis
  • Exhibit 4.11: Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
  • Exhibit 4.12: Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
  • Exhibit 4.13: Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
  • Exhibit 4.14: Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular Dystrophy
  • Exhibit 4.15: Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
  • Exhibit 4.16: Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
  • Exhibit 4.17: Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
  • Exhibit 4.18: Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
  • Exhibit 4.19: Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin Deficiency
  • Exhibit 4.20: Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic Arthritis
  • Exhibit 5.1: Total Number of Orphan Drugs in Phase I, II, III and NDA/BLA Clinical Programs
  • Exhibit 5.2: Number of Orphan Drugs (Oncology/Ex-Oncology) in Phase I, II, III and NDA/BLA Clinical Programs
  • Exhibit 5.3: Number of Therapies in Development for Rare Diseases
  • Exhibit 5.4: Top Selling Orphan Drugs Forecast to 2025
  • Exhibit 5.5: Top Orphan Drugs by Type and Mode of Action by 2025
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