表紙
市場調查報告書

焦點市場分析:X染色體脆折症候群

Market Spotlight: Fragile X Syndrome

出版商 Datamonitor Healthcare 商品編碼 858348
出版日期 內容資訊 英文 28 Pages
商品交期: 最快1-2個工作天內
價格
焦點市場分析:X染色體脆折症候群 Market Spotlight: Fragile X Syndrome
出版日期: 2020年08月17日內容資訊: 英文 28 Pages
簡介

根據需要特殊教育的兒童相關世代調查的結果,男兒4000人,女孩兒8000人有一人,X染色體脆折症候群 (FXS) 發病的可能性。複數的未來潛力治療藥物正在進行臨床實驗中,某些產品已進入II期。還有2015年至2020年之間已達成五項許可協議和資產收購交易,提高了對FXS治療劑開發的期望。就各國來看,美國在藥物開發方面處於領先地位。

本報告提供全球X染色體脆折症候群 (FXS) 治療藥的市場相關分析,彙整疾病概要和治療方法,目前發病情形,主要的已上市/開發平台醫藥品的概要,近來臨床實驗趨勢與今後的預測,近幾年的授權合約·資本交易的動向,專利趨勢,市場規模趨勢預測 (今後5年份)等資訊,為您概述為以下內容。

概要

主要的要點

疾病的背景

治療

流行病學

  • 前突然變異

開發平台醫藥品

近來的主要市場活動和分析師的見解

今後主要的活動

成功的可能性

授權合約·資產收購交易

母專利

臨床實驗環境

  • 臨床試驗贊助者一覽:各狀況
  • 臨床試驗贊助者一覽:各期

參考文獻

附錄

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: DMKC0191189

This Market Spotlight report covers the Fragile X Syndrome market, comprising key pipeline drugs, clinical trials, recent events and analyst opinion, upcoming events, probability of success, a discussion on disease epidemiology, and licensing and asset acquisition deals.

Key Takeaways

Based on cohorts of children with special educational needs, prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females.

The majority of industry-sponsored drugs in active clinical development for FXS are in Phase II, with only one drug in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA-A receptor, insulin-like growth factor-1 receptor, and potassium channels. The majority of these pipeline drugs are administered via the oral route.

The only high-impact upcoming event for drugs in the FXS space is topline Phase II trial results for BPN14770. The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 3.9%, and the average probability a drug advances from Phase III is 25%. Drugs, on average, take 8.2 years from Phase I to approval, compared to 9.8 years in the overall neurology space.

All clinical trials for FXS have been in Phase II, with no Phase III trials to date.

The US leads in terms of the number of FXS clinical trials globally.

No company has conducted more than one trial in the FXS space.

TABLE OF CONTENTS

CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

EPIDEMIOLOGY

  • Premutation

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

  • Zygel for FXS (June 30, 2020)
  • OV101 for FXS (May 7, 2020)

KEY UPCOMING EVENTS

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

  • Shionogi To Add CNS Critical Mass With Tetra Buyout

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase

BIBLIOGRAPHY

APPENDIX

LIST OF FIGURES

  • Figure 1: Overview of pipeline drugs for FXS in the US
  • Figure 2: Pipeline drugs for FXS, by company
  • Figure 3: Pipeline drugs for FXS, by drug type
  • Figure 4: Pipeline drugs for FXS, by classification
  • Figure 5: Zygel for FXS (June 30, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
  • Figure 6: OV101 for FXS (May 7, 2020): Phase II - ROCKET
  • Figure 7: Key upcoming events in FXS
  • Figure 8: Probability of success in the FXS pipeline
  • Figure 9: Clinical trials in FXS
  • Figure 10: Top 10 drugs for clinical trials in FXS
  • Figure 11: Top 10 companies for clinical trials in FXS
  • Figure 12: Trial locations in FXS
  • Figure 13: FXS trials status
  • Figure 14: FXS trials sponsors, by phase

LIST OF TABLES

  • Table 1: Pipeline drugs for FXS in the US
  • Table 2: Zygel for FXS (June 30, 2020)
  • Table 3: OV101 for FXS (May 7, 2020)
  • Table 4: Campral for FXS (June 25, 2019)