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脊髓性肌萎縮症 (SMA) :機會分析與預測

Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028

出版商 GlobalData 商品編碼 912099
出版日期 內容資訊 英文 88 Pages
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脊髓性肌萎縮症 (SMA) :機會分析與預測 Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028
出版日期: 2019年06月28日內容資訊: 英文 88 Pages
簡介

主要7個國家 (美國、法國、德國、義大利、西班牙、英國、日本)的脊髓性肌萎縮症 (SMA) 治療藥市場,預計將從2018年的15億美元,擴大到2028年66億美元,以年複合成長率 (CAGR) 16%的速度成長。

本報告提供主要7個國家的脊髓性肌萎縮症 (SMA)的治療藥市場相關調查分析,疾病概要,流行病學,目前治療選項,未滿足需求和機會,研究開發策略,開發平台評估等相關的系統性資訊。

第1章 目錄

第2章 摘要整理

第3章 簡介

第4章 疾病概要

  • 病因與病情生理
  • 病期分類

第5章 流行病學

  • 疾病的背景
  • 危險因素和合併症
  • 世界趨勢與歷史的趨勢
  • 預測手法
  • 脊髓性肌萎縮症 (SMA)的流行病學預測
  • 討論

第6章 目前治療選項

  • 概要

第7章 與未滿足需求機會評估

  • 概要
  • 新的作用機制與有利的給藥途徑
  • 疾病的早期診斷
  • 理解疾病的自然史的必要性
  • 進入障礙的消除

第8章 研究開發 (R&D) 策略

  • 概要
  • 臨床實驗設計

第9章 開發平台評估

  • 概要
  • 創新的初期階段的方法
  • 其他開發中的藥

第10章 開發平台評估分析

  • 主要開發平台藥物臨床基準
  • 主要開發平台藥物的商業基準
  • 競爭評估
  • 10年預測
    • 美國
    • EU5個國家
    • 日本

第11章 附錄

目錄
Product Code: GDHC096POA

The total Spinal Muscular Atrophy (SMA) market was estimated to be worth US$1.5bn across the seven major markets (7MM - US, France, Germany, Italy, Spain, UK and Japan) in 2018. This is expected to grow to US$6.6bn in 2028, at an impressive compound annual growth rate (CAGR) of 16%. This growth will be driven by the FDA approval of Novartis' Zolgensma for Spinal Muscular Atrophy (SMA) in May 2019, coupled with its anticipated approvals in the EU and Japan as well as the launch of other pipeline products during the forecast period.

Pipeline products with the potential to help address the root cause of Spinal Muscular Atrophy (SMA) by producing the missing protein in Spinal Muscular Atrophy (SMA) patients are the ones that have been met with enthusiasm among key opinion leaders (KOLs). Therapies that are being developed to help improve muscle functions in Spinal Muscular Atrophy (SMA) patients also are expected to play a pivotal role in patient care.

Until Zolgensma's recent FDA approval, Biogen's Spinraza was the only approved therapy for Spinal Muscular Atrophy (SMA) patients. While Spinraza has definitely reaped the benefits of first-to-market therapy for Spinal Muscular Atrophy (SMA), its need for a chronic dosage coupled with an intrathecal route of administration represents a logistical challenge for patients. Pharma companies have identified this gap as a potential area of improvement and their efforts are leading to the development of transformative therapies or ones with a more favorable route of administration. For example, Zolgensma is a gene-therapy that is proposed to be administered as a one-off infusion with the potential to cure the disease.

Launches of pipeline drugs with a more favorable route of administration, such as Roche's risdiplam and Novartis' branaplam, are expected to cause an immediate worry to Spinraza's dominant market position. A therapy that is required to be administered via an oral route represents a less intensive resource utilization scenario and for this reason, patients and physicians are likely to prefer such therapies over one that needs to be administered intrathecally.

While the development of clinical therapies for Spinal Muscular Atrophy (SMA) patients are picking up pace, there is another element that is critical for their success. Spinal Muscular Atrophy (SMA) is a rare indication and to off-set the costs of development of a drug that is approved for an indication with a small patient population inevitably means that these therapies are launched at a higher price-point. This is expected to be a topic of ongoing discussions between drug developers and funding agencies in order to help arrive at a value that is both realistic and sustainable for all stakeholders.

The latest report, "Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028" provides an overview of Spinal Muscular Atrophy (SMA), including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management. Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the SMA therapeutics market.

Key Questions Answered

  • What are the key unmet needs in SMA? What do KOLs identify as the key unmet needs? Will the products in the current pipeline fill these unmet needs?
  • What were the key SMA treatments in 2018?
  • When will the late stage pipeline products launch, and how will it affect drug sales and the overall SMA market in the 7MM?

Scope

  • Overview of SMA, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
  • Market Data including annualized SMA therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in fours patient segments (Type 1, 2, 3, and 4) forecast from 2018 to 2028.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the SMA therapeutics market.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for SMA therapy. The most promising candidates in Phase II and III development are profiled.
  • Analysis of the current and future market competition in the global SMA therapeutics market: Insightful review of the key industry drivers and barriers. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to:

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the global SMA therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global SMA therapeutics market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table of Contents

1 Table of Contents

  • 1.1 List of Tables
  • 1.2 List of Figures

2 Spinal Muscular Atrophy: Executive Summary

  • 2.1 Launch of Pipeline Products as Well as Newborn Screening Initiatives Are Expected to Drive the SMA Market Forward
  • 2.2 Distinct R&D Strategies Help Companies to Tackle SMA and Reap Benefits
  • 2.3 Environmental Unmet Needs Outweigh Clinical Unmet Needs
  • 2.4 What Do Physicians Think?

3 Introduction

  • 3.1 Catalyst
  • 3.2 Related Reports
  • 3.3 Upcoming Related Reports

4 Disease Overview

  • 4.1 Etiology and Pathophysiology
    • 4.1.1 Etiology
    • 4.1.2 Pathophysiology
  • 4.2 Classification of Spinal Muscular Atrophy

5 Epidemiology

  • 5.1 Disease Background
  • 5.2 Risk Factors and Comorbidities
  • 5.3 Global and Historical Trends
  • 5.4 Forecast Methodology
    • 5.4.1 Sources
    • 5.4.2 Forecast Assumptions and Methods
    • 5.4.3 Forecast Assumptions and Methods - Diagnosed Prevalent Cases of SMA
  • 5.5 Epidemiological Forecast for SMA (2018-2028)
    • 5.5.1 Diagnosed Prevalent Cases of SMA
    • 5.5.2 Sex-Specific Diagnosed Prevalent Cases of SMA
    • 5.5.3 Diagnosed Prevalent Cases of SMA by Type
  • 5.6 Discussion
    • 5.6.1 Epidemiological Forecast Insight
    • 5.6.2 Limitations of Analysis
    • 5.6.3 Strengths of Analysis

6 Current Treatment Options

  • 6.1 Overview

7 Unmet Needs and Opportunity Assessment

  • 7.1 Overview
  • 7.2 Novel Mechanisms of Action and Favorable Routes of Administration
  • 7.3 Early Diagnosis of Disease
  • 7.4 Need to Understand the Natural History of Disease
  • 7.5 Removal of Access Barriers

8 R&D Strategies

  • 8.1 Overview
    • 8.1.1 Development of a Novel Gene Therapy that Addresses the Cause of 5q SMA and Requires One-Time Administration
    • 8.1.2 Development of Oral Agents that can Modulate RNA Splicing
    • 8.1.3 Development of Therapeutic Agents that Can Increase Strength and Muscle Function
  • 8.2 Clinical Trials Design
    • 8.2.1 Outcome Measures and Trial Designs
    • 8.2.2 Patient Population, Recruitment, and Retention
    • 8.2.3 Trial Duration

9 Pipeline Assessment

  • 9.1 Overview
  • 9.2 Innovative Early-Stage Approaches
  • 9.3 Other Drugs in Development

10 Pipeline Valuation Analysis

  • 10.1 Clinical Benchmark of Key Pipeline Drugs
  • 10.2 Commercial Benchmark of Key Pipeline Drugs
  • 10.3 Competitive Assessment
  • 10.4 Top-Line 10-Year Forecast
    • 10.4.1 US
    • 10.4.2 5EU
    • 10.4.3 Japan

11 Appendix

  • 11.1 Bibliography
  • 11.2 Abbreviations
  • 11.3 Methodology
    • 11.3.1 Forecasting Methodology
    • 11.3.2 Diagnosed Prevalent Cases of SMA Patients
    • 11.3.3 Percent Drug-Treated Patients
    • 11.3.4 Drugs Included in Each Therapeutic Class
    • 11.3.5 Launch and Patent Expiry Dates
    • 11.3.6 General Pricing Assumptions
    • 11.3.7 Individual Drug Assumptions
    • 11.3.8 Generic Erosion
    • 11.3.9 Pricing of Pipeline Agents
  • 11.4 Primary Research - KOLs Interviewed for This Report
    • 11.4.1 KOLs
    • 11.4.2 Payers
  • 11.5 Primary Research - Prescriber Survey
  • 11.6 About the Authors
    • 11.6.1 Analyst
    • 11.6.2 Therapy Area Director
    • 11.6.3 Epidemiologist
    • 11.6.4 Reviewers
    • 11.6.5 Global Director of Therapy Analysis and Epidemiology
    • 11.6.6 Global Head and EVP of Healthcare Operations and Strategy
  • 11.7 About GlobalData
  • 11.8 Contact Us
  • 11.9 Disclaimer

List of Tables

  • Table 1: Spinal Muscular Atrophy: Key Metrics in the Seven Markets
  • Table 2: Clinical Classification of SMA
  • Table 3: Risk Factors and Comorbidities for SMA
  • Table 4: Treatment Guidelines for Spinal Muscular Atrophy
  • Table 5: Leading Treatments for Spinal Muscular Atrophy, 2019
  • Table 6: Comparison of Therapeutic Classes in Development for SMA, 2018-2028
  • Table 7: Innovative Early-Stage Approaches for SMA, 2019
  • Table 8: Drugs in Development for SMA, 2019
  • Table 9: Clinical Benchmark of Key Pipeline Drugs - SMA
  • Table 10: Commercial Benchmark of Key Pipeline Drugs - SMA
  • Table 11: Key Events Impacting Global Sales for Spinal Muscular Atrophy, 2018-2028
  • Table 12: Spinal Muscular Atrophy Market - Global Drivers and Barriers, 2018-2028
  • Table 13: Key Historical and Projected Launch Dates for SMA
  • Table 14: Key Historical and Projected Patent Expiry Dates for SMA
  • Table 15: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

  • Figure 1: Global Sales Forecast by Country for Spinal Muscular Atrophy in 2018 and 2028
  • Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Spinraza
  • Figure 3: Difference Between SMN1 and SMN2 Ge