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市場調查報告書

裘馨氏肌肉萎縮症(DMD)治療藥的全球市場 - 分析與預測:各治療方法(突變抑制,外顯子跳躍 、類固醇療法) 2018年∼2023年

Duchenne Muscular Dystrophy (DMD) Drugs Market Size, Share & Trends Analysis Report By Therapeutic Approach (Mutation Suppression, Exon Skipping, Steroid Therapy) And Segment Forecasts, 2018 - 2023

出版商 Grand View Research, Inc. 商品編碼 726424
出版日期 內容資訊 英文 77 Pages
商品交期: 2-3個工作天內
價格
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裘馨氏肌肉萎縮症(DMD)治療藥的全球市場 - 分析與預測:各治療方法(突變抑制,外顯子跳躍 、類固醇療法) 2018年∼2023年 Duchenne Muscular Dystrophy (DMD) Drugs Market Size, Share & Trends Analysis Report By Therapeutic Approach (Mutation Suppression, Exon Skipping, Steroid Therapy) And Segment Forecasts, 2018 - 2023
出版日期: 2018年08月02日內容資訊: 英文 77 Pages
簡介

全球裘馨氏肌肉萎縮症(DMD)市場在2018年到2023年間預測將以41.3%的年複合成長率發展,到2023年市場規模將成長到41億1,000萬美元。對突變特別型治療藥,及標靶治療的偏好高漲,對市場有利的政府的配合措施等成為這個市場的主要成長要素。

本報告提供全球裘馨氏肌肉萎縮症(DMD)市場相關調查,提供市場概要,各主要7個國家的流行病學,開發平台及後期臨床開發階段的治療藥概要,市場趨勢,競爭情形,主要企業的簡介等全面性資訊。

目錄

第1章 調查方法

  • 資訊採購
  • 資料分析

第2章 摘要整理

第3章 疾病的底層塗料和流行病學

  • 病的底層塗料
  • 流行病學
  • 市場(美國主要7個國家、日本、歐盟5國的)盛行率與發病率(現值)
  • 市場(美國主要7個國家、日本、歐盟5國的)盛行率與發病率(預測值)

第4章 市場概要

  • 簡介與市場概要
  • 成長要素與課題
  • 交易環境
  • 定價與給付制度
  • SWOT分析

第5章 開發平台資訊

  • 開發平台概要
  • 後期開發平台治療藥
  • 創新的新藥的簡介

第6章 企業簡介

  • Sarepta Therapeutics
  • PTC Therapeutics
  • Santhera Pharmaceuticals
  • Italfarmaco
  • Catabasis

第7章 市場預測

  • 市場未來展望
  • 勝者和敗者
  • 新興企業
目錄
Product Code: GVR-2-68038-423-9

The global duchenne muscular dystrophy (DMD) drugs market size is expected to reach USD 4.11 billion by 2023, according to a new report by Grand View Research, Inc., expanding at a CAGR of 41.3% during the forecast period. Several factors such as emergence of mutation-specific therapies, growing target population, and favorable government initiatives are driving the market.

The DMD therapeutics market has only three approved products - Exondys51, Translarna, and Emflaza. Increasing uptake of mutation-targeted therapies is likely to boost the sales of branded drugs. However, stringent regulatory procedures and lack of standardized protocol for determination of clinical efficacy are key challenges for the market.

Exon-skipping platform is estimated to hold about 45.0% of the market share by 2023, driven by increasing adoption of Exondys51 and impending approval of golodirsen and casimersen. Mutation-specific therapies, such as Translarna, are anticipated to face limited adoption due to premium pricing. Associated adverse effects of steroid therapy make them a less favored treatment option.

The DMD drugs market is projected to become intensely competitive in medium- to long-term. The sector has witnessed delays and denials of several key products by the U.S. FDA due to insufficient trials. Raxone (by Santhera) and Givinostat (by Italfarmaco) remain the most strategically significant R&D pipeline assets for the DMD market, with good possibility of regulatory approval during the forecast period.

Launch of late-stage pipeline products is poised to alter the DMD therapeutic landscape in the near future. Several novel mechanisms of action, such as NF-KB inhibition, myostatin inhibition, and gene therapy are under investigation. Other drug classes, such as exon-skipping and mutation-suppression, are expected to gain traction over the forecast period, supported by rising adoption of these therapeutics.

Milo Biotechnology is one of the first companies to venture into gene therapy for DMD. Company's investigational therapy uses an adeno-associated virus as a myostatin inhibitor and is in early stages of development. Wave Life Science's pipeline product is likely to perform 25 times better in terms of clinical efficacy than the existing exon-skipping drugs.

Further key findings from the report suggest:

Exon-skipping platform dominated the therapeutic approaches in 2017, capturing nearly 40.0% of the market. Exondys51 is estimated to be the leading drug for DMD by 2023

The U.S. dominated the regional market share and is projected to maintain its lead through 2023, owing to rising disease incidence and anticipated launch of promising pipeline candidates

Mutation-suppressive therapeutics and steroids are projected to witness healthy growth due to increasing adoption of Translarna and Emflaza

Translarna received conditional approval in Europe in 2014 for treatment of nonsense mutation DMD patients who are in ambulatory state and are 5 years or older. Exondys51 and Emflaza have not been approved for DMD treatment in Europe

Sarepta, PTC, Santhera, Italfarmaco, and Catabasis are some of the key players operating in this arena

Sarepta is poised to lead the market by 2023 due to higher adoption of Exondys51 as well as anticipated launch of pipeline candidates

Table of Contents

Chapter 1 Research Methodology

  • 1.1 Information Procurement
  • 1.2 Information or Data Analysis
    • 1.2.1 Market formulation & validation

Chapter 2 Executive Summary

Chapter 3 Disease Primer and Epidemiology

  • 3.1 Disease Primer
    • 3.1.1 Symptoms
    • 3.1.2 Progression of Disease
  • 3.2 Epidemiology
  • 3.3 Current Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)
  • 3.4 Forecast Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)

Chapter 4 Global Duchenne Muscular Dystrophy Drugs Market Overview

  • 4.1 Introduction and Market Overview
    • 4.1.1 Segmentation, by therapeutic approach
      • 4.1.1.1 Mutation suppression
      • 4.1.1.2 Exon skipping
      • 4.1.1.3 Steroid therapy
    • 4.1.2 Segmentation, by major markets
      • 4.1.2.1 U.S.
      • 4.1.2.2 EU5
      • 4.1.2.3 Japan
    • 4.1.3 Market size and forecast
    • 4.1.4 Sales performance
    • 4.1.5 Market share distribution
    • 4.1.6 Market dynamics among leading brands
  • 4.2 Drivers and Challenges
  • 4.3 Deals Landscape (2013-2018)
  • 4.4 Pricing and Reimbursement
  • 4.5 SWOT Analysis

Chapter 5 Duchenne Muscular Dystrophy Drugs Market: Pipeline Intelligence

  • 5.1 Pipeline Landscape
    • 5.1.1 Drugs in development
    • 5.1.2 Key R&D trends
      • 5.1.2.1 Corticosteroids
      • 5.1.2.2 NF-KB Inhibitor
      • 5.1.2.3 Anti-myostatin
      • 5.1.2.4 Gene Therapy
      • 5.1.2.5 Exon Skipping Platform
  • 5.2 Late-Stage Pipeline
  • 5.3 Profile of Disruptive Drugs
    • 5.3.1 Vamorolone (VBP15)
    • 5.3.2 SMT-C1100 (ezutromid)
    • 5.3.3 CAT-1004 (Edasalonexent)
    • 5.3.4 WVE-210201
    • 5.3.5 AAV1-FS344

Chapter 6 Company Profiles

  • 6.1 Sarepta Therapeutics
    • 6.1.1 Company overview
    • 6.1.2 Current product portfolio
    • 6.1.3 Product forecast sales up to 2023
    • 6.1.4 Company - key news flow
    • 6.1.5 Pipeline view
    • 6.1.6 Pipeline forecast
    • 6.1.7 Catalysts and event calendar
    • 6.1.8 SWOT analysis
  • 6.2 PTC Therapeutics
    • 6.2.1 Company overview
    • 6.2.2 Current product portfolio
    • 6.2.3 Product forecast sales up to 2023
    • 6.2.4 Company - key news flow
    • 6.2.5 Pipeline view
    • 6.2.6 Pipeline forecast
    • 6.2.7 Catalysts and event calendar
    • 6.2.8 SWOT Analysis
  • 6.3 Santhera Pharmaceuticals
    • 6.3.1 Company overview
    • 6.3.2 Current product portfolio
    • 6.3.3 Product forecast sales up to 2023
    • 6.3.4 Company - key news flow
    • 6.3.5 Pipeline view
    • 6.3.6 Pipeline forecast
    • 6.3.7 Catalysts and event calendar
    • 6.3.8 SWOT analysis
  • 6.4 Italfarmaco
    • 6.4.1 Company overview
    • 6.4.2 Current product portfolio
    • 6.4.3 Company - key news flow
    • 6.4.4 Pipeline view
    • 6.4.5 Pipeline forecast
    • 6.4.6 Catalysts and event calendar
    • 6.4.7 SWOT analysis
  • 6.5 Catabasis
    • 6.5.1 Company overview
    • 6.5.2 Current product portfolio
    • 6.5.3 Company - key news flow
    • 6.5.4 Pipeline view
    • 6.5.5 Pipeline forecast
    • 6.5.6 Catalysts and event calendar
    • 6.5.7 SWOT analysis

Chapter 7 Market Outlook

  • 7.1 What the Future Holds
  • 7.2 Winners and Losers
  • 7.3 Emerging Companies
  • 7.4 The Road Ahead

List of Tables

  • TABLE 1 Duchenne Muscular Dystrophy Prevalence and Incidence - 2017
  • TABLE 2 Forecast Duchenne Muscular Dystrophy Prevalence and Incidence, 2017 - 2023
  • TABLE 3 Duchenne Muscular Dystrophy Drugs Market Size and Forecast (in USD million)
  • TABLE 4 Geographic Sales Performance, by Seven Major Markets (in USD million)
  • TABLE 5 Duchenne Muscular Dystrophy Drugs Market, by Therapeutic Approach (in USD million)
  • TABLE 6 Sales Forecast of Approved Treatments (in USD million)
  • TABLE 7 Duchenne Muscular Drugs Sales Forecast, by Company (in USD million)
  • TABLE 8 Duchenne Muscular Dystrophy Drugs Market Share, by Company (in USD million)
  • TABLE 9 Duchenne Muscular Dystrophy Deals Landscape (2013 - 2018)
  • TABLE 10 Duchenne Muscular Dystrophy Drugs in Development
  • TABLE 11 Late-Stage Duchenne Muscular Dystrophy Pipeline
  • TABLE 12 Pipeline Forecast - Global Duchenne Muscular Drugs Market
  • TABLE 13 Profile of Disruptive Drug: Vamorolone (VBP15)
  • TABLE 14 Profile of Disruptive Drug: SMT-C1100 (ezutromid)
  • TABLE 15 Profile of Disruptive Drug: CAT-1004 (edasalonexent)
  • TABLE 16 Profile of Disruptive Drug: WVE-210201
  • TABLE 17 Profile of Disruptive Drug: AAV1-FS344
  • TABLE 18 Product Portfolio: Exondys 51
  • TABLE 19 Exondys 51 Sales Forecast through 2023
  • TABLE 20 Sarepta Pipeline View
  • TABLE 21 Sarepta - Upcoming Events and Catalysts
  • TABLE 22 Product Portfolio: Translarna
  • TABLE 23 Product Portfolio: Emflaza
  • TABLE 24 Translarna Sales Forecast through 2023
  • TABLE 25 Emflaza Sales Forecast through 2023
  • TABLE 26 PTC Pipeline View
  • TABLE 27 PTC - Upcoming Events and Catalysts
  • TABLE 28 Product Portfolio: Raxone
  • TABLE 29 Raxone Sales Forecast through 2023
  • TABLE 30 Santhera Pipeline View
  • TABLE 31 Santhera - Upcoming Events and Catalysts
  • TABLE 32 Product Portfolio: Givinostat
  • TABLE 33 Italfarmaco Pipeline View
  • TABLE 34 Italfarmaco - Upcoming Events and Catalysts
  • TABLE 35 Product Portfolio: CAT-1004
  • TABLE 36 Catabasis Pipeline View
  • TABLE 37 Catabasis - Upcoming Events and Catalysts

List of Figures

  • FIG. 1 Market Research Process
  • FIG. 2 Information Procurement
  • FIG. 3 Primary Research Pattern
  • FIG. 4 Market Research Approaches
  • FIG. 5 Value Chain Based Sizing & Forecasting
  • FIG. 6 QFD Modelling for Market Share Assessment
  • FIG. 7 Progression of Duchenne Muscular Dystrophy
  • FIG. 8 Current Prevalence Across Seven Major Markets - 2017
  • FIG. 9 Current Incidence Across Seven Major Markets - 2017
  • FIG. 10 Market segmentation & scope
  • FIG. 11 Geographic Sales Performance, by Seven Major Markets 2017
  • FIG. 12 Duchenne Muscular Dystrophy Drugs Market, by Therapeutic Approach
  • FIG. 13 Duchenne Muscular Dystrophy Sales Trend, by Therapeutic Approach
  • FIG. 14 Duchenne Muscular Dystrophy Drugs Market Shares, by Drug Class (2017A-2023E)
  • FIG. 15 U.S. Duchenne Muscular Dystrophy Drugs Market, by Therapeutic Approach
  • FIG. 16 EU5 Duchenne Muscular Dystrophy Drugs Market, by Therapeutic Approach
  • FIG. 17 Japan Duchenne Muscular Dystrophy Drugs Market, by Therapeutic Approach
  • FIG. 18 Duchenne Muscular Dystrophy Drugs Market Share, by Company (2017A-2023E)
  • FIG. 19 Market Trends & Outlook
  • FIG. 20 Market Driver Relevance Analysis (Current & Future Impact)
  • FIG. 21 Market Restraint Relevance Analysis (Current & Future Impact)
  • FIG. 22 SWOT Analysis (Duchenne Muscular Dystrophy Drugs Market)
  • FIG. 23 Pipeline Assets by Phase
  • FIG. 24 Exon Skipping and Estimated DMD Risk
  • FIG. 25 SWOT Analysis (Sarepta Therapeutics)
  • FIG. 26 SWOT Analysis (PTC Therapeutics)
  • FIG. 27 SWOT Analysis (Santhera Therapeutics)
  • FIG. 28 SWOT Analysis (Italfarmaco)
  • FIG. 29 SWOT Analysis (Catabasis)
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