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市場調查報告書

法布瑞氏症:開發中產品分析

Fabry Disease - Pipeline Review, H2 2019

出版商 Global Markets Direct 商品編碼 200448
出版日期 內容資訊 英文 112 Pages
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法布瑞氏症:開發中產品分析 Fabry Disease - Pipeline Review, H2 2019
出版日期: 2019年11月25日內容資訊: 英文 112 Pages
簡介

法布瑞氏症是遺傳性疾病,是由於體內血管壁稱作globotriaosylceramide(GL-3)的特定脂肪物質異常堆積而發病。症候有疼痛,腹瀉,噁心,腎臟障礙,耳鳴,心律不整,心臟瓣膜症等,治療方法為使用酵素補充療法等。

本報告提供法布瑞氏症的治療藥開發情形調查分析,提供您開發中產品概要,臨床實驗的各階段產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等資訊。

簡介

  • 調查範圍

法布瑞氏症概要

治療藥的開發

  • 開發中產品的概要
  • 開發中產品比較分析

法布瑞氏症:企業開發中的治療藥

法布瑞氏症:開發中產品概況

  • 後期階段的產品
  • 臨床階段的產品
  • 初期階段的產品

法布瑞氏症:企業開發中的產品

法布瑞氏症的治療藥的開發企業

  • Actelion Ltd
  • Alexion Pharmaceuticals Inc
  • Amicus Therapeutics, Inc.
  • Genzyme Corporation
  • Greenovation Biotech GmbH
  • ISU ABXIS Co.,Ltd.
  • JCR Pharmaceuticals Co., Ltd.
  • Neuraltus Pharmaceuticals, Inc.
  • Pharming Group N.V.
  • Protalix BioTherapeutics, Inc.
  • Sangamo BioSciences, Inc.
  • Shire Plc

法布瑞氏症:治療藥的評估

  • 單劑產品
  • 組合產品
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

法布瑞氏症:暫停中的計劃

法布瑞氏症:開發中止的產品

法布瑞氏症:產品開發的里程碑

  • 主要消息和新聞稿

附錄

圖表

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目錄
Product Code: GMDHC11568IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 2019, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.

Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused due to mutations in GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A (this enzyme is responsible for breaking down of globotriaosylceramide). Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Fabry Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Fabry Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fabry Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 5, 1, 7 and 2 respectively. Similarly, the Universities portfolio in Unknown stages comprises 1 molecules, respectively.

Fabry Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Fabry Disease (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Fabry Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Fabry Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Fabry Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Fabry Disease (Genetic Disorders)

Reasons to buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Fabry Disease (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Fabry Disease (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

  • List of Tables
  • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Fabry Disease - Overview
  • Fabry Disease - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Fabry Disease - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Fabry Disease - Companies Involved in Therapeutics Development
    • 4D Molecular Therapeutics Inc
    • Abeona Therapeutics Inc
    • Amicus Therapeutics Inc
    • AVROBIO Inc
    • Biosidus SA
    • Chiesi Farmaceutici SpA
    • Freeline Therapeutics Ltd
    • Genzyme Corp
    • Greenovation Biotech GmbH
    • Idorsia Pharmaceutical Ltd
    • Moderna Therapeutics Inc
    • Pharming Group NV
    • Resverlogix Corp
    • Sangamo Therapeutics Inc
    • Sigilon Therapeutics Inc
    • UniQure NV
    • Yuhan Corp
  • Fabry Disease - Drug Profiles
    • 4D-310 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • agalsidase beta biosimilar - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AMT-190 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • apabetalone - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AVRRD-01 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • FLT-190 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Fabry Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate GLA for Fabry Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • lucerastat - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • migalastat hydrochloride - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MOSS-AGAL - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • mRNA-3630 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pegunigalsidase alfa - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PGN-005 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Sig-007 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ST-920 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • venglustat malate - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • YHNCE-10 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Fabry Disease - Dormant Projects
  • Fabry Disease - Discontinued Products
  • Fabry Disease - Product Development Milestones
    • Featured News & Press Releases
      • Nov 18, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici announce successful pre-BLA meeting with FDA for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
      • Oct 17, 2019: Protalix BioTherapeutics announces positive 12-month interim data from the BRIDGE phase III open label switch-over study of Pegunigalsidase Alfa for the treatment of Fabry Disease
      • Sep 25, 2019: Protalix and Chiesi close enrolment in Phase III Fabry disease trial
      • Aug 02, 2019: Amicus Therapeutics receives marketing authorization for galafold (migalastat) for fabry disease in Argentina
      • Jul 15, 2019: AVROBIO announces 87% substrate reduction in first kidney biopsy and additional positive data from clinical trials of AVR-RD-01 investigational gene therapy in Fabry Disease
      • Jul 02, 2019: Handok releases Fabry disease treatment
      • Jun 17, 2019: Protalix Biotherapeutics completes enrollment in the phase III bright clinical trial of Pegunigalsidase Alfa (PRX 102) for the treatment of Fabry disease
      • Jun 06, 2019: Protalix BioTherapeutics and Chiesi Farmaceutici to apply for accelerated approval of pegunigalsidase alfa for the treatment of Fabry Disease in the United States
      • May 30, 2019: 4D Molecular Therapeutics announces presentation of preclinical data at the 6th International Update on Fabry Disease and provides clinical update
      • May 02, 2019: uniQure announces new preclinical data on fabry disease in oral presentations at the 22nd ASGCT Annual Meeting
      • May 01, 2019: Avrobio to incorporate plato platform into FAB-201 Phase II trial
      • Apr 15, 2019: uniQure to present data on Gene Therapy for Fabry Disease at ASGCT 2019
      • Mar 18, 2019: Resverlogix proudly announces funding for phase 2 trial evaluating Apabetalone in Pulmonary Arterial Hypertension led by Quebec Heart and Lung Institute - Laval University researcher
      • Feb 20, 2019: Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
      • Feb 06, 2019: AVROBIO announces updated clinical data from ongoing phase 1 and phase 2 studies for AVR-RD-01 gene therapy for fabry disease
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Table 1: Number of Products under Development for Fabry Disease, H2 2019
  • Table 2: Number of Products under Development by Companies, H2 2019
  • Table 3: Number of Products under Development by Universities/Institutes, H2 2019
  • Table 4: Products under Development by Companies, H2 2019
  • Table 5: Products under Development by Companies, H2 2019 (Contd..1), H2 2019
  • Table 6: Products under Development by Universities/Institutes, H2 2019
  • Table 7: Number of Products by Stage and Target, H2 2019
  • Table 8: Number of Products by Stage and Mechanism of Action, H2 2019
  • Table 9: Number of Products by Stage and Route of Administration, H2 2019
  • Table 10: Number of Products by Stage and Molecule Type, H2 2019
  • Table 11: Fabry Disease - Pipeline by 4D Molecular Therapeutics Inc, H2 2019
  • Table 12: Fabry Disease - Pipeline by Abeona Therapeutics Inc, H2 2019
  • Table 13: Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2019
  • Table 14: Fabry Disease - Pipeline by AVROBIO Inc, H2 2019
  • Table 15: Fabry Disease - Pipeline by Biosidus SA, H2 2019
  • Table 16: Fabry Disease - Pipeline by Chiesi Farmaceutici SpA, H2 2019
  • Table 17: Fabry Disease - Pipeline by Freeline Therapeutics Ltd, H2 2019
  • Table 18: Fabry Disease - Pipeline by Genzyme Corp, H2 2019
  • Table 19: Fabry Disease - Pipeline by Greenovation Biotech GmbH, H2 2019
  • Table 20: Fabry Disease - Pipeline by Idorsia Pharmaceutical Ltd, H2 2019
  • Table 21: Fabry Disease - Pipeline by Moderna Therapeutics Inc, H2 2019
  • Table 22: Fabry Disease - Pipeline by Pharming Group NV, H2 2019
  • Table 23: Fabry Disease - Pipeline by Resverlogix Corp, H2 2019
  • Table 24: Fabry Disease - Pipeline by Sangamo Therapeutics Inc, H2 2019
  • Table 25: Fabry Disease - Pipeline by Sigilon Therapeutics Inc, H2 2019
  • Table 26: Fabry Disease - Pipeline by UniQure NV, H2 2019
  • Table 27: Fabry Disease - Pipeline by Yuhan Corp, H2 2019
  • Table 28: Fabry Disease - Dormant Projects, H2 2019
  • Table 29: Fabry Disease - Discontinued Products, H2 2019

List of Figures

  • Figure 1: Number of Products under Development for Fabry Disease, H2 2019
  • Figure 2: Number of Products under Development by Companies, H2 2019
  • Figure 3: Number of Products by Targets, H2 2019
  • Figure 4: Number of Products by Stage and Targets, H2 2019
  • Figure 5: Number of Products by Mechanism of Actions, H2 2019
  • Figure 6: Number of Products by Stage and Mechanism of Actions, H2 2019
  • Figure 7: Number of Products by Routes of Administration, H2 2019
  • Figure 8: Number of Products by Stage and Routes of Administration, H2 2019
  • Figure 9: Number of Products by Molecule Types, H2 2019
  • Figure 10: Number of Products by Stage and Molecule Types, H2 2019
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