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市場調查報告書

孤兒藥 (罕見疾病用醫藥品) 市場分析:至2025年的預測

Orphan Drug Market Analysis and Forecast to 2025

出版商 Kelly Scientific Publications 商品編碼 979793
出版日期 內容資訊 英文 197 Pages
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孤兒藥 (罕見疾病用醫藥品) 市場分析:至2025年的預測 Orphan Drug Market Analysis and Forecast to 2025
出版日期: 2020年12月01日內容資訊: 英文 197 Pages
簡介

本報告提供全球孤兒藥 (罕見疾病用醫藥品) 市場相關調查分析,在這個領域最普及治療方法為焦點,目前市場規模與預測,市場詳細內容,主要的產業企業與趨勢,各地區的考察與趨勢等相關的系統性資訊。

目錄

第1章 摘要整理

第2章 美國 (FDA) 、歐洲 (EMA) 的孤兒藥的認證

  • 創業投資隨著時間的推移而增加
  • 治療領域占主導地位
  • 孤兒藥指定趨勢
  • 現有的治療藥的轉用
  • 日本的核准趨勢
  • 韓國的核准趨勢
  • 中國的核准趨勢

第3章 罕見疾病市場分析

  • 主要的孤兒藥市場
  • 孤兒低分子市場分析
  • 孤兒抗體市場分析
  • 孤兒基因治療市場分析
  • 孤兒細胞療法市場分析
  • 孤兒寡核苷酸療法市場分析
  • 罕見疾病市場地理明細
  • 孤兒藥市場:各適應症、各疾病類型
  • 主要的市場企業
    • Bristol-Myers Squibb
    • Roche/Genentech
    • Alexion
    • J&J/Janssen Biotech
    • Biogen
    • Novartis
    • AstraZeneca
    • Eisai
    • Takeda
    • Abbvie
    • Gilead
    • Pfizer
    • Vertex Pharmaceuticals
    • Argenix

第4章 臨床試驗的開發平台醫藥品:各症狀,各介入,各階段

  • 低分子和多發性骨髓瘤
  • 黑色素瘤的基因治療、細胞療法
  • ADA-SCID的基因治療、細胞療法
  • RPE65遺傳基因變異視網膜
  • 稀少的眼科疾病與孤兒藥
  • 脊髓性肌萎縮症和孤兒藥
  • 鐮狀細胞貧血症和孤兒藥
  • A、B型血友病的基因治療、細胞療法
  • 囊狀纖維化症的階段III
  • 囊狀纖維化症的基因治療、細胞療法
  • 胰臟癌症的CAR-T細胞治療藥
  • 多發性骨髓瘤的CAR-T細胞治療藥
  • 裘馨氏肌肉萎縮症的基因治療、細胞療法
  • 罕見疾病的寡核苷酸療法
  • 紅血球合成性紫質症的治療藥
  • 格林-巴利症候群的治療藥
  • 硬皮症的治療藥
  • Alpha1-抗胰蛋白酶缺乏症的治療藥
  • 幼年特發性關節炎的治療藥

第5章 開發平台治療藥分析

第6章 未來預測

目錄

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

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