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市場調查報告書
商品編碼
858348

焦點市場分析:X染色體脆折症候群

Market Spotlight: Fragile X Syndrome

出版日期: | 出版商: Datamonitor Healthcare | 英文 30 Pages | 商品交期: 最快1-2個工作天內

價格
  • 全貌
  • 簡介
  • 目錄
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

目錄
Product Code: DMKC0191189

Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment. FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP). FMRP is required for normal brain development, and individuals with FXS do not make this protein. Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP

Key Takeaways

Based on cohorts of children with special educational needs, the 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 two drugs 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, GABA-B receptor, 11-beta-hydroxylase, 11 betahydroxysteroid dehydrogenase type 1, and potassium channels. The majority of these pipeline drugs are administered via the oral route.

The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 2.7%, and the average probability a drug advances from Phase III is 20%. Drugs, on average, take 12.4 years from Phase I to approval, compared to 10.0 years in the overall neurology space.

The distribution of clinical trials across Phase I-IV indicates that the majority of trials for FXS have been in the early and midphases of development, with 77% of trials in Phase I-II, and only 23% in Phase III-IV.

The US has a substantial lead in the number of FXS clinical trials globally. France and Spain lead the major European markets, while Israel has the top spot in Asia.

Clinical trial activity in the FXS space is dominated by completed trials. Seaside Therapeutics and Novartis have the highest number of completed clinical trials for FXS, with four trials each.

Seaside Therapeutics leads industry sponsors with the highest overall number of clinical trials for FXS, followed by Novartis.

TABLE OF CONTENTS

CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

EPIDEMIOLOGY

  • Premutation

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

  • BPN14770 for FXS (November 2, 2020)
  • Zygel for FXS (October 15, 2020)
  • Zygel for FXS (June 30, 2020)
  • OV101 for FXS (May 7, 2020)

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: BPN14770 for FXS (November 2, 2020): Phase II - Adults
  • Figure 6: Zygel for FXS (October 15, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
  • Figure 7: Zygel for FXS (June 30, 2020): Phase III - CONNECT-FX (US, AUS, NZ)
  • Figure 8: OV101 for FXS (May 7, 2020): Phase II - ROCKET
  • Figure 9: Probability of success in the neurodevelopmental disorders pipeline
  • Figure 10: Clinical trials in FXS
  • Figure 11: Top 10 drugs for clinical trials in FXS
  • Figure 12: Top 10 companies for clinical trials in FXS
  • Figure 13: Trial locations in FXS
  • Figure 14: FXS trials status
  • Figure 15: FXS trials sponsors, by phase

LIST OF TABLES

  • Table 1: Pipeline drugs for FXS in the US
  • Table 2: BPN14770 for FXS (November 2, 2020)
  • Table 3: Zygel for FXS (October 15, 2020)
  • Table 4: Zygel for FXS (June 30, 2020)
  • Table 5: OV101 for FXS (May 7, 2020)