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脊髄性筋萎縮症 (SMA) :機会分析と予測

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

発行 GlobalData 商品コード 912099
出版日 ページ情報 英文 88 Pages
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脊髄性筋萎縮症 (SMA) :機会分析と予測 Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028
出版日: 2019年06月28日 ページ情報: 英文 88 Pages
概要

主要7ヶ国 (米国、フランス、ドイツ、イタリア、スペイン、英国、日本) の脊髄性筋萎縮症 (SMA) 治療薬市場は、2018年の15億米ドルから、2028年には66億米ドルまで拡大する見込みで、CAGR (複合年間成長率) で、16%の成長が予測されています。

当レポートでは、主要7ヶ国の脊髄性筋萎縮症 (SMA) の治療薬市場について調査分析し、疾患の概要、疫学、現在の治療選択肢、アンメットニーズと機会、研究開発戦略、パイプライン評価などについて、体系的な情報を提供しています。

第1章 目次

第2章 エグゼクティブサマリー

第3章 イントロダクション

第4章 疾患の概要

  • 病因と病態生理
  • 病期分類

第5章 疫学

  • 疾患の背景
  • 危険因子と併存疾患
  • 世界動向と歴史的動向
  • 予測手法
  • 脊髄性筋萎縮症 (SMA) の疫学予測
  • 議論

第6章 現在の治療選択肢

  • 概要

第7章 アンメットニーズと機会評価

  • 概要
  • 新しい作用機序と好ましい投与経路
  • 疾患の早期診断
  • 疾患の自然史を理解する必要性
  • 参入障壁の除去

第8章 研究開発 (R&D) 戦略

  • 概要
  • 治験設計

第9章 パイプライン評価

  • 概要
  • 革新的な初期段階のアプローチ
  • その他の開発中の薬

第10章 パイプライン評価分析

  • 主要パイプライン薬の臨床ベンチマーク
  • 主要パイプライン薬の商業ベンチマーク
  • 競合評価
  • 10ヶ年予測
    • 米国
    • EU5ヶ国
    • 日本

第11章 付録

図表

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
目次
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
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