表紙:注目市場の分析:脆弱X症候群
市場調査レポート
商品コード
858348

注目市場の分析:脆弱X症候群

Market Spotlight: Fragile X Syndrome

出版日: | 発行: Datamonitor Healthcare | ページ情報: 英文 30 Pages | 納期: 即日から翌営業日

価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=114.84円
注目市場の分析:脆弱X症候群
出版日: 2021年06月10日
発行: Datamonitor Healthcare
ページ情報: 英文 30 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 目次
概要

特殊教育が必要な児童に関するコホート調査の結果によると、男児は4000人、女児は8000人に一人が、脆弱X症候群 (FXS) を発症する可能性があります。複数の将来有望な治療薬の治験が進行しており、第II相に達した製品もいくつかあります。また、2015~2020年に5件ものライセンス契約や資産買収取引が締結されており、FXS治療薬の開発に対する期待が高まっています。国別では、米国が薬剤開発の動きを主導しています。

当レポートでは、世界の脆弱X症候群 (FXS) 治療薬の市場について分析し、疾患の概要や治療方法、現在の発症状況、主な上市済み/パイプライン医薬品の概略、昨今の治験動向と今後の見通し、近年のライセンス契約・資本取引の動き、特許動向、市場規模の動向見通し (今後5年間分)、といった情報を取りまとめてお届けいたします。

概要

主な要点

疾患の背景

治療

疫学

  • 前突然変異

パイプライン医薬品

昨今の市場の主な出来事と、アナリストの見解

今後の主なイベント

成功の可能性

ライセンス契約・資産買収取引

母体特許

治験環境

  • 治験依頼者の一覧:状況別
  • 治験依頼者の一覧:相 (フェーズ) 別

参考文献

付録

目次
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)