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軟骨無形成症治療薬:パイプライン製品の分析

Achondroplasia - Pipeline Review, H2 2019

発行 Global Markets Direct 商品コード 484624
出版日 ページ情報 英文 57 Pages
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軟骨無形成症治療薬:パイプライン製品の分析 Achondroplasia - Pipeline Review, H2 2019
出版日: 2019年11月25日 ページ情報: 英文 57 Pages
概要

当レポートでは、軟骨無形成症の治療薬開発パイプラインの現況と最新アップデートによる各開発段階の比較分析、企業や研究機関によって開発中の治療薬、治療薬の評価、休止中・中止されたプロジェクトなどに関する情報を提供しています。

イントロダクション

  • 調査範囲

軟骨無形成症 - 概要

軟骨無形成症 - 治療薬の開発

  • パイプラインの概要
  • 企業別のパイプライン動向
  • 企業で開発中の製品

軟骨無形成症 - 治療薬の評価

  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

軟骨無形成症の治療薬開発に従事している企業

軟骨無形成症 - 薬剤プロファイル

軟骨無形成症 - 休止中のプロジェクト

軟骨無形成症 - 製品開発のマイルストーン

  • 主なニュースとプレスリリース

付録

図表

List of Tables

  • Table 1: Number of Products under Development for Achondroplasia, H2 2019
  • Table 2: Number of Products under Development by Companies, H2 2019
  • Table 3: Products under Development by Companies, H2 2019
  • Table 4: Number of Products by Stage and Target, H2 2019
  • Table 5: Number of Products by Stage and Mechanism of Action, H2 2019
  • Table 6: Number of Products by Stage and Route of Administration, H2 2019
  • Table 7: Number of Products by Stage and Molecule Type, H2 2019
  • Table 8: Achondroplasia - Pipeline by Ascendis Pharma A/S, H2 2019
  • Table 9: Achondroplasia - Pipeline by BioMarin Pharmaceutical Inc, H2 2019
  • Table 10: Achondroplasia - Pipeline by Daiichi Sankyo Co Ltd, H2 2019
  • Table 11: Achondroplasia - Pipeline by Pfizer Inc, H2 2019
  • Table 12: Achondroplasia - Pipeline by PhaseBio Pharmaceuticals Inc, H2 2019
  • Table 13: Achondroplasia - Pipeline by QED Therapeutics Inc, H2 2019
  • Table 14: Achondroplasia - Pipeline by Ribomic Inc, H2 2019
  • Table 15: Achondroplasia - Dormant Projects, H2 2019

List of Figures

  • Figure 1: Number of Products under Development for Achondroplasia, 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 Top 10 Targets, H2 2019
  • Figure 5: Number of Products by Mechanism of Actions, H2 2019
  • Figure 6: Number of Products by Stage and Top 10 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
目次
Product Code: GMDHC11555IDB

Summary:

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

Achondroplasia is a bone growth disorder that causes disproportionate dwarfism. This is caused by mutations in the FGFR3 gene. Symptoms include decreased muscle tone, apnea, hydrocephalus, short arms and legs, disproportionately large head compared to the body and kyphosis. Treatment includes growth hormones.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Achondroplasia - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Achondroplasia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 1, 4 and 1 respectively.

Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal Disorders).
  • The pipeline guide reviews pipeline therapeutics for Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal 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 Achondroplasia (Musculoskeletal 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
  • Achondroplasia - Overview
  • Achondroplasia - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Products under Development by Companies
  • Achondroplasia - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Achondroplasia - Companies Involved in Therapeutics Development
    • Ascendis Pharma A/S
    • BioMarin Pharmaceutical Inc
    • Daiichi Sankyo Co Ltd
    • Pfizer Inc
    • PhaseBio Pharmaceuticals Inc
    • QED Therapeutics Inc
    • Ribomic Inc
  • Achondroplasia - Drug Profiles
    • ACP-015 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
      • ASB-20123 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Fusion Protein to Agonize NPR2 for Achondroplasia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • infigratinib phosphate - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RBM-007 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • TA-100 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • TA-46 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • vosoritide - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Achondroplasia - Dormant Projects
  • Achondroplasia - Product Development Milestones
    • Featured News & Press Releases
      • Nov 14, 2019: BioMarin announces cumulative additional height gain of 9.0 cm over 54 months versus natural history in children with Achondroplasia treated with Vosoritide in phase 2 study
      • Nov 06, 2019: BioMarin to highlight innovative development pipeline including vosoritide at R&D Day on November 14th
      • Oct 17, 2019: QED and parent company BridgeBio announce preclinical data supporting tolerability and activity of low-dose infigratinib in treating Achondroplasia
      • Sep 12, 2019: Ascendis Pharma announces presentations on Achondroplasia and TransCon CNP at International Skeletal Dysplasia Society Meeting
      • Jul 18, 2019: Ascendis Pharma announces filing of Investigational New Drug (IND) application for initiation of a global phase 2 trial for TransCon CNP in Children with Achondroplasia
      • Jun 18, 2019: BioMarin announces New England Journal of medicine publishes vosoritide phase 2 study showing sustained annualized growth up to 42 months in children with Achondroplasia
      • Feb 28, 2019: Ascendis Pharma announces orphan drug designation granted for TransCon CNP as treatment for achondroplasia
      • Jan 07, 2019: BioMarin highlights Vosoritide at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
      • Nov 28, 2018: Ascendis Pharma announces positive preliminary phase 1 data for TransCon CNP
      • Nov 07, 2018: BioMarin provides update on Vosoritide at R&D day in New York
      • Nov 01, 2018: BioMarin provides update on vosoritide at R&D Day on November 7th in New York
      • Jun 21, 2018: Therachon Announces Start of Natural History Study in Children with Achondroplasia
      • Jun 14, 2018: BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
      • May 08, 2018: Ascendis Pharma Announces Dosing of First Subjects in Phase 1 Trial of TransCon CNP
      • Feb 14, 2018: Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer
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