表紙:脆弱X症候群(2021年~2030年):市場規模および動向レポート(疫学およびパイプライン分析、競合評価、アンメットニーズ、臨床試験戦略、予測を含む)
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1066873

脆弱X症候群(2021年~2030年):市場規模および動向レポート(疫学およびパイプライン分析、競合評価、アンメットニーズ、臨床試験戦略、予測を含む)

Fragile X Syndrome Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021 - 2030

出版日: | 発行: GlobalData | ページ情報: 英文 60 Pages | 納期: 即納可能 即納可能とは

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本日の銀行送金レート: 1USD=135.61円
脆弱X症候群(2021年~2030年):市場規模および動向レポート(疫学およびパイプライン分析、競合評価、アンメットニーズ、臨床試験戦略、予測を含む)
出版日: 2022年02月18日
発行: GlobalData
ページ情報: 英文 60 Pages
納期: 即納可能 即納可能とは
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概要

脆弱X症候群(FXS)の市場規模は、4つのパイプライン薬剤の参入により、2020年から2030年にかけて大きな伸びを示すと予想されます。特に、高価格のパイプライン薬剤であるザイゲル、ザトルミラスト、トロフィネティドの売上は、最もインパクトのある促進要因となります。第I相および第II相の開発段階にある製品がFXSパイプラインの89.7%を占め、第III相試験での開発は限られており、現在開発中の医薬品は1つだけです。

当レポートは脆弱X症候群の世界市場を調査し、FXSの概要(疫学、症状、診断、疾患管理など)、2020年から2030年までの3つの患者セグメント(0~11歳の小児、12~17歳の青年、18歳以上の成人)におけるFXS治療薬市場の年間収益、患者あたりの治療費、治療使用パターンなどの予測、競合情勢、市場の特徴、アンメットニーズ、臨床試験のマッピング、FXS治療薬市場に対するこれらの要因の影響など、体系的な情報を提供しています。

目次

目次

第1章 エグゼクティブサマリー

  • エグゼクティブサマリー

第2章 病気の概要

  • 脆弱X症候群の概要

第2章 脆弱X症候群のSWOT分析

  • 脆弱X症候群の分類

第3章 疫学

  • FXSの診断された一般的な症例、男性と女性(2020年~2030年)
  • ソースと調査手法

第4章 現在の治療選択肢

  • 治療パラダイム
  • 現在の治療選択肢
  • 製品プロファイル:SSRI(フルオキセチン、セルトラリン、シタロプラム、エスシタロプラム)
  • 製品プロファイル:その他の抗うつ薬(ベンラファキシン、ブプロピオン、トラゾドン)
  • 製品プロファイル:精神刺激薬(メチルフェニデート塩酸塩)
  • 製品プロファイル:非定型抗精神病薬(アリピプラゾール、リスペリドン)
  • 製品プロファイル:抗けいれん薬(ラモトリジン、カルバマゼピン、バルプロ酸ナトリウム)
  • 製品プロファイル:降圧抗不安薬(クロニジン、グアンファシン)
  • 製品プロファイル:ベンゾジアゼピン(クロナゼパム、ジアゼパム)
  • 製品プロファイル:リチウム(炭酸リチウム)

第5章 アンメットニーズと機会

  • 脆弱X症候群におけるアンメットニーズ

第6章 研究開発戦略

  • FXSでの取引の動向

第7章 パイプライン評価

  • 脆弱X症候群パイプラインの概要
  • 後期パイプラインエージェント
  • 製品プロファイル:ZynerbaPharmaceuticalsのZygel
  • 製品プロファイル:TetraTherapeuticsのzatolmilast
  • 製品プロファイル:ConfluencePharmaceuticalsのAcamprosate Calcium SR
  • 製品プロファイル:Neuren PharmaceuticalsおよびAcadia PharmaceuticalsのTrofinetide

第8章 市場の見通し

  • 脆弱X症候群の市場予測
  • 市場促進要因と障壁

第9章 付録

  • 参考文献
  • 1次調査:KOL情報
  • 著者について

第10章 お問い合わせ

目次
Product Code: GDHCOA014

Fragile X syndrome (FXS) (ICD-10: Q99.2) is an X-linked genetic condition and is the most common cause of inherited learning disability. FXS is caused by expansion of a cytosine, guanine, and guanine (CGG) trinucleotide repeats in the 5' untranslated region of the fragile X mental retardation 1 (FMR1) gene. This expansion results in transcriptional silencing of the FMR1 gene with a subsequent reduction or absence of fragile X mental retardation protein (FMRP) expression. FMRP is an important mediator in dendritic maturity and synaptic plasticity.

FXS is a lifelong disorder associated with a spectrum of developmental and behavioral issues, including psychiatric symptoms (anxiety, attention deficit hyperactivity disorder [ADHD], and poor eye contact) and neurologic symptoms (seizures and movement disorders). Other common features of the syndrome include characteristic physical attributes (large and prominent ears, long face, and flat feet) as well as deafness, due to the high recurrence of ear infections. The severity of these symptoms is dependent on the amount of FMRP protein that is produced, which in turn is dependent on both the number of CGG repeats as well as the degree of methylation in the FMR1 gene. As females have two X chromosomes and thus two copies of the FMR1 gene, females often have milder symptoms than males.

The two clinically and commercially relevant segments considered for the classification of FXS in this report are segmented into three groups by age: pediatric patients aged 0-11 years, adolescent patients aged 12-17, and adult patients aged 18+.

This report covers 2MM (US and Germany) and provides an Excel-based forecast model for the Fragile X Syndrome market through 2030.

Key Highlights

  • Forecasts include two countries
  • Forecasts cover three time points: base year, 5-year, and 10-year
  • There are currently no approved therapies available for FXS, so prescribed treatments are exclusively off-label products that target individual symptoms of the disease. The most frequently used drug classes are: selective serotonin reuptake inhibitors (SSRIs), psychostimulants, antipsychotic agents, anticonvulsant agents, and antihypertensive anxiolytics.
  • The current treatment options that target FXS symptoms of irritability and aggression are often sedating and diminish patients' interaction with their environment. This prevents disruptive behavior but negatively affects the patients' quality of life. There is a need for interventions that suppress aggressive behavior without side effects.
  • Products in Phase I and II stages of development account for 89.7% of the FXS pipeline, while limited development is seen in Phase III trials with only one drug currently in development.
  • The FXS market will exhibit significant growth between 2020 and 2030, driven by the entry of four pipeline agents. Notably, the sales of high-priced pipeline agents, Zygel, zatolmilast, and trofinetide represent the most impactful drivers of growth.

Scope

  • Overview of FXS, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized FXS therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in three patient segments (pediatric patients aged 0-11 years, adolescent patients aged 12-17, and adult patients aged 18+) forecast from 2020 to 2030.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications of these factors for the FXS therapeutics market.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for FXS treatment. The most promising candidates in Phase III and Phase IIb development are profiled.
  • Analysis of the current and future market competition in the global FXS therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to Buy

  • 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 FXS therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global FXS therapeutics market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing 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

Table of Contents

1 Executive Summary

  • 1.1 Executive Summary

2 Disease Overview

  • 2.1 Overview of Fragile X Syndrome

2.2 Fragile X Syndrome SWOT Analysis

  • 2.3 Classification of Fragile X Syndrome

3 Epidemiology

  • 3.1 Diagnosed Prevalent Cases of FXS, Men and Women, 2020-30
  • 3.2 Sources and Methodology

4 Current Treatment Options

  • 4.1 Treatment Paradigm
  • 4.2 Current Treatment Options
  • 4.3 Product Profile: SSRIs (fluoxetine, sertraline, citalopram, escitalopram)
  • 4.4 Product Profile: Other Antidepressants (venlafaxine, bupropion, trazadone)
  • 4.5 Product Profile: Psychostimulants (methylphenidate hydrochloride)
  • 4.6 Product Profile: Atypical Antipsychotics (aripiprazole, risperidone)
  • 4.7 Product Profile: Anticonvulsants (lamotrigine, carbamazepine, sodium valproate)
  • 4.8 Product Profile: Antihypertensive Anxiolytics (clonidine, guanfacine)
  • 4.9 Product Profile: Benzodiazepines (clonazepam, diazepam)
  • 4.10 Product Profile: Lithium (lithium carbonate)

5 Unmet Needs and Opportunities

  • 5.1 Unmet Needs in Fragile X Syndrome

6 R&D Strategies

  • 6.1 Trends in Deal-Making in FXS
  • 6.2 Trends in Deal-Making in FXS

7 Pipeline Assessment

  • 7.1 Fragile X Syndrome Pipeline Overview
  • 7.2 Late-Stage Pipeline Agents
  • 7.3 Product Profile: Zynerba Pharmaceuticals' Zygel
  • 7.4 Product Profile: Tetra Therapeutics' zatolmilast
  • 7.5 Product Profile: Confluence Pharmaceuticals' Acamprosate Calcium SR
  • 7.6 Product Profile: Neuren Pharmaceuticals' and Acadia Pharmaceuticals' Trofinetide

8 Market Outlook

  • 8.1 Fragile X Syndrome Market Forecast
  • 8.2 Market Drivers and Barriers

9 Appendix

  • 9.1 Bibliography
  • 9.2 Primary Research: KOL Information
  • 9.3 About the Authors

10 Contact Us