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市場調查報告書

裘馨氏肌肉萎縮症:機會及市場分析

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

出版商 GlobalData 商品編碼 333932
出版日期 內容資訊 英文 154 Pages
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裘馨氏肌肉萎縮症:機會及市場分析 OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019
出版日期: 2015年04月01日 內容資訊: 英文 154 Pages
簡介

裘馨氏肌肉萎縮症 (DMD) 是新生男孩約每3500人中有1人發生的稀少遺傳性疾病,具有漸進性肌肉退變,無法行走,及到20歲後半為止死亡的特徵。2015年PTC Therapeutics Translarna 在美國及EU5個國家預定銷售,外顯子略過治療藥 - BioMarin/Prosensa drisapersen 及 Sarepta Therapeutics eteplirsen 也將各在2015年、2016年預定銷售,治療形勢變化,促進DMD市場成長。醫藥品的主要6個國家的市場 (美國、法國、德國、義大利、西班牙、英國) 的DMD市值2014年為820萬美金,預計今後以160.5%的年複合成長率發展,2019年劇增到約9億9,000萬美元。

本報告提供醫藥品的主要6個國家的市場 (美國、法國、德國、義大利、西班牙、英國) 的裘馨氏肌肉萎縮症 (DMD) 市場形勢的相關調查、最有前途的後期開發平台藥物與其銷售與未來的治療形勢帶來的影響、DMD市場上龐大的未滿足需求與留下的機會等資料彙整,為您概述為以下內容。

第1章 目錄

第2章 簡介

第3章 疾病概要

  • 病因、病理學
  • 預後、症狀
  • 診斷

第4章 流行病學

  • 疾病的背景
  • 危險因素和共生病症
  • 全球趨勢
  • 預測手法
    • 利用之資訊來源
    • 未利用之資訊來源
    • 預測的前提條件與手法
  • 裘馨氏肌肉萎縮症 (DMD) 的流行病學預測
    • DMD的診斷病例數 (全年齡層)
    • DMD的診斷病例數 (5-13歲)
    • 各年齡的DMD診斷病例數: (全年齡層)
    • 年齡調整DMD診斷病例數
  • DMD的無意義突變相關流行病學預測
  • DMD的外顯子略過療法的適用性相關流行病學預測
  • 貝克氏肌肉萎縮症 (BMD) 的流行病學預測
    • BMD的診斷病例數 (全年齡層)
    • 各年齡的BMD診斷病例數 (全年齡層) 年齡調整BMD診斷病例數
  • 議論
    • 流行病學預測相關考察
    • 分析的限制
    • 分析的優勢

第5章 目前治療選項

  • 概要
    • 裘馨氏肌肉萎縮症 (DMD)
    • 貝克氏肌肉萎縮症 (BMD)
  • 產品簡介:領導品牌
    • 皮質類固醇
    • Translarna (ataluren)

第6章 未滿足需求的評估、機會分析

  • 概要
  • 疾病緩解疾病療法的必要性
    • 未滿足需求
    • 差距分析
    • 機會
  • 診斷的延遲
  • DMD臨床實驗設計的高變動率
  • 朝向後續外顯子略過分子的認證的管制途徑的必要性

第7章 R & D策略

  • 概要
  • 目前臨床實驗設計
  • 未來臨床實驗設計

第8章 開發平台評估

  • 概要
  • 臨床開發的有前途藥物
  • 創新的早期階段方法

第9章 開發平台核定分析

  • 主要的開發平台藥物臨床基準
  • 主要的開發平台藥物的商業基準
  • 競爭評估
  • 銷售額的5年預測
    • 美國
    • EU5個國家

第10章 附錄

圖表

目錄
Product Code: GDHC038POA

Duchenne muscular dystrophy (DMD) is a rare genetic disorder affecting approximately 1 in 3,500 newborn boys and is characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. There are no marketed pharmacological therapies that are indicated for DMD. The current standard of care treatment involves generic corticosteroids which are used to marginally prolong muscle function but do not offer preventative treatment. The anticipated launch of PTC Therapeutics' Translarna in 2015 in the US and 5EU, followed by the 6MM launch of Exon-skipping therapies- BioMarin/Prosensa's drisapersen and Sarepta Therapeutics' eteplirsen, in 2015 and 2016, respectively, are set to change the treatment landscape and drive growth in the DMD market. GlobalData estimates that the uptake of Translarna and exon-skipping therapies will be very high due to the lack of effective disease-modifying therapies for DMD. GlobalData estimates that the DMD market was valued at $8.2m across the 6MM in 2014, and it is expected to sharply increase to approximately $990.0m in 2019, at a Compound Annual Growth Rate (CAGR) of 160.5%.

Highlights

Key Questions Answered

  • How will the DMD market landscape change within the 2014-2019 forecast periods in the 6MM?
  • What are the most promising late-stage pipeline drugs and how will their launch shape the future treatment landscape in the DMD market?
  • How do the clinical and commercial attributes of late-stage pipeline drugs compare to one another and against existing treatment options?
  • Which patient population(s) are most likely to be targeted by late-stage pipeline drugs?
  • What are the significant unmet needs and remaining opportunities in the DMD market?

Key Findings

  • Due to strong demand from patient community and clinicians, high uptake is expected of pipeline drugs for DMD that are anticipated to launch during the 2014-2019 forecast period.
  • Mutation-specific therapies are expected to emerge as the major R&D breakthrough and future market drivers for DMD.
  • With ongoing research, large pharmaceutical companies, small biotechs, and research institutions have discovered a multitude of therapeutic strategies to treat DMD. There are 19 ongoing clinical trials in DMD with 11 molecules in Phase II and eight molecules in Phase I.
  • The lack of consensus over clinical trial design and endpoints for the development of DMD therapies continues to be a barrier to clinical development of new drugs.
  • The clinical stage pipeline is mainly focussed on ambulatory DMD patients with specific mutations, leaving a large unaddressed population and vast opportunity for developers to deliver new therapies, and for continued growth in the DMD market beyond 2019.

Scope

  • Overview of DMD, including epidemiology, etiology, pathophysiology, symptoms and current treatment options
  • Annualized DMD therapeutics market revenue, annual cost of therapies and forecasts for five years to 2019.
  • Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the DMD therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including nonsense mutation readthrough inducer, exon-skipping therapies, synthetic electron transporter, PDE5 inhibitor.
  • Analysis of the current and future market competition in the US and five major EU DMD therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.

Reasons to buy

  • Identify the unmet needs and remaining opportunities in the DMD therapeutics market.
  • Develop business strategies by understanding the trends shaping and driving the US and five major EU DMD therapeutics market.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Assess the clinical and commercial viability of promising pipeline products.
  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
  • Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU DMD therapeutics market in future.

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Introduction

  • 2.1. Catalyst
  • 2.2. Related Reports

3. Disease Overview

  • 3.1. Etiology and Pathophysiology
    • 3.1.1. Etiology
    • 3.1.2. Pathophysiology
  • 3.2. Prognosis and Symptoms
  • 3.3. Diagnosis

4. Epidemiology

  • 4.1. Disease Background
  • 4.2. Risk Factors and Comorbidities
  • 4.3. Global Trends
  • 4.4. Forecast Methodology
    • 4.4.1. Sources Used
    • 4.4.2. Sources Not Used
    • 4.4.3. Forecast Assumptions and Methods
  • 4.5. Epidemiological Forecast for DMD (2013-2023)
    • 4.5.1. Diagnosed Prevalent Cases of DMD (All Ages)
    • 4.5.2. Diagnosed Prevalent Cases of DMD (Ages 5-13 Years)
    • 4.5.3. Age-Specific Diagnosed Prevalent Cases of DMD (All Ages)
    • 4.5.4. Age-Standardized Diagnosed Prevalence of DMD
  • 4.6. Epidemiological Forecast for Nonsense Mutations in DMD (2013-2023)
  • 4.7. Epidemiological Forecast for the Applicability of Exon-Skipping Therapies in DMD (2013-2023)
    • 4.7.1. Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy (All Ages)
    • 4.7.2. Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy (All Ages)
    • 4.7.3. Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy (All Ages)
    • 4.7.4. Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy (All Ages)
  • 4.8. Epidemiological Forecast for BMD (2013-2023)
    • 4.8.1. Diagnosed Prevalent Cases of BMD (All Ages)
    • 4.8.2. Age-Specific Diagnosed Prevalent Cases of BMD (All Ages)
    • 4.8.3. Age-Standardized Diagnosed Prevalence of BMD
  • 4.9. Discussion
    • 4.9.1. Epidemiological Forecast Insight
    • 4.9.2. Limitations of the Analysis
    • 4.9.3. Strengths of the Analysis

5. Current Treatment Options

  • 5.1. Overview
    • 5.1.1. Duchenne Muscular Dystrophy
    • 5.1.2. Becker Muscular Dystrophy
  • 5.2. Product Profiles - Major Brands
    • 5.2.1. Corticosteroids (Prednisone and Deflazacort - Numerous Generic and Brand Names)
    • 5.2.2. Translarna (ataluren)

6. Unmet Needs Assessment and Oppportunity Analysis

  • 6.1. Overview
  • 6.2. Need for Disease-Modifying Therapies
    • 6.2.1. Unmet Need
    • 6.2.2. Gap Analysis
    • 6.2.3. Opportunity
  • 6.3. Delayed Diagnosis
    • 6.3.1. Unmet Need
    • 6.3.2. Gap Analysis
    • 6.3.3. Opportunity
  • 6.4. High Variation in Design of DMD Clinical Trials
    • 6.4.1. Unmet Need
    • 6.4.2. Gap Analysis
    • 6.4.3. Opportunity
  • 6.5. Need for Regulatory Pathway for Approval of Follow-On Exon-Skipping Molecules
    • 6.5.1. Unmet Need
    • 6.5.2. Gap Analysis
    • 6.5.3. Opportunity

7. R&D Strategies

  • 7.1. Overview
    • 7.1.1. Follow-On and Multi Exon-Skipping Molecules
    • 7.1.2. Forming Alliances with Patient Advocacy Groups
    • 7.1.3. Partnering with Pharmaceutical Companies to Increase Marketing Resources
  • 7.2. Current Clinical Trial Design
    • 7.2.1. Current Methods for Assessing Clinical Efficacy and Their Limitations
    • 7.2.2. Current Trial Design of Key Pipeline Products
    • 7.2.3. Patient Exclusion Issues in Current Trial Designs
  • 7.3. Future Clinical Trial Design
    • 7.3.1. Dystrophin-Positive Biomarkers as Surrogate Clinical Endpoints
    • 7.3.2. Need for Measuring Clinical Outcome and Endpoints Based on Disease Onset
    • 7.3.3. Maximizing Inclusion of Patient Populations in Studies

8. Pipeline Assessment

  • 8.1. Overview
  • 8.2. Promising Drugs in Clinical Development
    • 8.2.1. Drisapersen
    • 8.2.2. Eteplirsen
    • 8.2.3. Catena/Raxone (idebenone)
    • 8.2.4. Tadalafil
  • 8.3. Innovative Early-Stage Approaches
    • 8.3.1. Follow-On Exon-Skipping Therapies
    • 8.3.2. Utrophin Activation
    • 8.3.3. Myostatin Inhibition
    • 8.3.4. Gene Therapy
    • 8.3.5. Targeting Cellular Pathways and Other Early-Stage Approaches

9. Pipeline Valuation Analysis

  • 9.1. Clinical Benchmark of Key Pipeline Drugs
  • 9.2. Commercial Benchmark of Key Pipeline Drugs
  • 9.3. Competitive Assessment
  • 9.4. Top-Line Five-Year Forecast
    • 9.4.1. US
    • 9.4.2. 5EU

10. Appendix

  • 10.1. Bibliography
  • 10.2. Abbreviations
  • 10.3. Methodology
  • 10.4. Forecasting Methodology
    • 10.4.1. Diagnosed DMD Patients
    • 10.4.2. Percent Drug-Treated Patients
    • 10.4.3. Drugs Included in Each Therapeutic Class
    • 10.4.4. Launch and Patent Expiry Dates
    • 10.4.5. General Pricing Assumptions
    • 10.4.6. Individual Drug Assumptions
    • 10.4.7. Generic Erosion
    • 10.4.8. Pricing of Pipeline Agents
  • 10.5. Physicians and Specialists Included in this Study
  • 10.6. About the Authors
    • 10.6.1. Author
    • 10.6.2. Reviewers
    • 10.6.3. Epidemiologist
    • 10.6.4. Global Head of Healthcare
  • 10.7. About GlobalData
  • 10.8. Disclaimer

List of Tables

  • Table 1: Symptoms of DMD
  • Table 2: Risk Factors and Comorbidities for DMD and BMD
  • Table 3: Global Estimates for the Diagnosed Prevalence of DMD and BMD
  • Table 4: MD STARnet Case Definition Criteria for DMD and BMD
  • Table 5: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD in the 6MM
  • Table 6: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of BMD in the 6MM
  • Table 7: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD with Nonsense Mutations in the 6MM
  • Table 8: Sources of Epidemiological Data Used for the Forecast for the Diagnosed Prevalent Cases of DMD Eligible for Exon-Skipping Therapies (Exons 51, 45, 53, or 44) in the 6MM
  • Table 9: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, Select Years 2013-2023
  • Table 10: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, Select Years 2013-2023
  • Table 11: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N (Row %), 2013
  • Table 12: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, Select Years 2013-2023
  • Table 13: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023
  • Table 14: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023
  • Table 15: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023
  • Table 16: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, Select Years 2013-2023
  • Table 17: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, Select Years 2013-2023
  • Table 18: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N (Row %), 2013
  • Table 19: Current Treatment Options for Duchenne Muscular Dystrophy
  • Table 20: Product Profile - Corticosteroids (Generics - Prednisone, Deflazacort)
  • Table 21: Corticosteroids SWOT Analysis, 2014
  • Table 22: Product Profile - Translarna
  • Table 23: Translarna SWOT Analysis, 2014
  • Table 24: Unmet Need and Opportunity in Duchenne Muscular Dystrophy
  • Table 25: Clinical Trial Design of Key Pipeline Drugs for DMD, January 2015
  • Table 26: Clinical Outcome Measures Based on Age and Onset of DMD
  • Table 27: DMD - Late-Stage Pipeline, February 2015
  • Table 28: Product Profile - Drisapersen
  • Table 29: Drisapersen SWOT Analysis, 2014
  • Table 30: Product Profile - Eteplirsen
  • Table 31: Eteplirsen SWOT Analysis, 2014
  • Table 32: Product Profile - Catena
  • Table 33: Catena SWOT Analysis, 2014
  • Table 34: Product Profile - Tadalafil
  • Table 35: Tadalafil SWOT Analysis, 2014
  • Table 36: Early-Stage Pipeline Products in DMD, February 2015
  • Table 37: Clinical Benchmark of Key Pipeline Drugs for DMD
  • Table 38: Commercial Benchmark of Key Pipeline Drugs for DMD
  • Table 39: Top-Line Sales Forecasts ($m) for DMD, 2014-2019
  • Table 40: Key Events Impacting Sales for DMD, 2014-2019
  • Table 41: DMD Market - Drivers and Barriers, 2014-2019
  • Table 42: Key Launch Dates, DMD, 2014-2019
  • Table 43: Key Patent Expiries, 2014-2019

List of Figures

  • Figure 1: Schematic Representation of Dystrophin and Associated Proteins in Muscle.
  • Figure 2: Patient Flow for the Epidemiological Forecast of DMD in the 6MM
  • Figure 3: 6MM, Diagnosed Prevalent Cases of DMD, Males, All Ages, N, 2013-2023
  • Figure 4: 6MM, Diagnosed Prevalent Cases of DMD, Males, Ages 5-13 Years, N, 2013-2023
  • Figure 5: 6MM, Age-Specific Diagnosed Prevalent Cases of DMD, Men, N, 2013
  • Figure 6: 6MM, Age-Standardized Diagnosed Prevalence of DMD (Cases per 100,000 Population), All Ages, Men, 2013
  • Figure 7: 6MM, Diagnosed Prevalent Cases of DMD with Nonsense Mutations, Males, All Ages, N, 2013-2023
  • Figure 8: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 51 Skipping Therapy, Males, All Ages, N, 2013-2023
  • Figure 9: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 45 Skipping Therapy, Males, All Ages, N, 2013-2023
  • Figure 10: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 53 Skipping Therapy, Males, All Ages, N, 2013-2023
  • Figure 11: 6MM, Diagnosed Prevalent Cases of DMD Eligible for an Exon 44 Skipping Therapy, Males, All Ages, N, 2013-2023
  • Figure 12: 6MM, Diagnosed Prevalent Cases of BMD, Males, All Ages, N, 2013-2023
  • Figure 13: 6MM, Age-Specific Diagnosed Prevalent Cases of BMD, Men, N, 2013
  • Figure 14: 6MM, Age-Standardized Diagnosed Prevalence of BMD (Cases per 100,000 Population), All Ages, Men, 2013
  • Figure 15: Competitive Assessment of Key Pipeline Drugs for DMD, 2014-2019
  • Figure 16: Sales for DMD by Region, 2014-2019
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