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発作性夜間ヘモグロビン尿症:パイプライン製品の分析

Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017

発行 Global Markets Direct 商品コード 269903
出版日 ページ情報 英文 90 Pages
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発作性夜間ヘモグロビン尿症:パイプライン製品の分析 Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017
出版日: 2017年04月28日 ページ情報: 英文 90 Pages
概要

発作性夜間ヘモグロビン(血色素)尿症(PNH)は希少な疾患で、赤血球が通常より早く破壊されます。腹痛、腰痛、暗色尿、あざができやすい/出血しやすい、頭痛、息切れといった症状が見られます。素因は、年齢、肥満、ホルモン療法です。治療には、手術、化学療法、放射線療法などが含まれます。

当レポートでは、発作性夜間ヘモグロビン尿症に対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向、最新ニュースとプレスリリースなどについて、体系的な情報を提供しています。

目次

イントロダクション

発作性夜間ヘモグロビン尿症の概要

治療薬の開発

  • パイプライン製品:概要

開発中の治療薬:企業別

パイプライン製品の概要

  • 治験段階の製品
  • 初期段階の製品

開発中の製品:企業別

治療薬の開発に従事している企業

  • Achillion Pharmaceuticals, Inc.
  • Akari Therapeutics, Plc
  • Alexion Pharmaceuticals Inc
  • Alnylam Pharmaceuticals, Inc.
  • Amgen Inc.
  • Amyndas Pharmaceuticals LLC
  • Apellis Pharmaceuticals Inc
  • Epirus Biopharmaceuticals, Inc.
  • Novartis AG
  • Omeros Corporation
  • Ra Pharmaceuticals, Inc.
  • Regenesance BV
  • The International Biotechnology Center (IBC) Generium

治療薬の評価

  • 単独療法の場合
  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

薬剤のプロファイル

休止状態のプロジェクト

開発が中止された製品

製品開発のマイルストーン

  • 最新ニュースとプレスリリース

付録

図表

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC9187IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017, provides an overview of the Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline landscape.

Paroxysmal nocturnal hemoglobinuria is a rare disease in which red blood cells break down earlier than normal. Symptoms include abdominal pain, back pain, dark urine, easy bruising or bleeding, headache and shortness of breath. The predisposing factors include age, obesity and hormone therapy. Treatment includes surgery, chemotherapy and radiation therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Paroxysmal Nocturnal Hemoglobinuria 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, 6, 2, 13 and 1 respectively.

Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological 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 Paroxysmal Nocturnal Hemoglobinuria (Hematological 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

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Paroxysmal Nocturnal Hemoglobinuria - Overview
    • Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Products under Development by Companies
  • Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development
    • Achillion Pharmaceuticals Inc
    • Akari Therapeutics Plc
    • Alexion Pharmaceuticals Inc
    • Alnylam Pharmaceuticals Inc
    • Amgen Inc
    • Apellis Pharmaceuticals Inc
    • F. Hoffmann-La Roche Ltd
    • ISU ABXIS Co Ltd
    • Novartis AG
    • NovelMed Therapeutics Inc
    • Omeros Corp
    • Ra Pharmaceuticals Inc
    • Regeneron Pharmaceuticals Inc
    • Regenesance BV
  • Paroxysmal Nocturnal Hemoglobinuria - Drug Profiles
    • ACH-4471 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ALN-CC5 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ALXN-1210 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AMY-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • APL-2 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • APL-9 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Coversin - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • eculizumab biosimilar - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • eculizumab biosimilar - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • GNR-045 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NM-9405 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OMS-721 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OMS-906 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RA-101348 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RA-101495 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Regenemab - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • REGN-3918 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RG-6107 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Inhibit Factor D for PNH and Dense Deposit Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit Factor D for Paroxysmal Nocturnal Hemoglobinuria and Membranoproliferative Glomerulonephritis Type II - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Synthetic Peptide to Inhibit Complement C5 for Autoimmune Disorders, Atypical Hemolytic Uremic Syndrome, Central Nervous System and Paroxysmal Nocturnal Hemoglobinuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • tesidolumab - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects
  • Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products
  • Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones
    • Featured News & Press Releases
      • Apr 24, 2017: Akari Therapeutics Demonstrates Positive Response with Coversin in Ongoing Phase 2 PNH Trial and in Additional Clinical Targets
      • Apr 06, 2017: Achillion Announces Initiation of Patient Dosing in Phase 2 Study of ACH-4471 for Paroxysmal Nocturnal Hemoglobinuria
      • Mar 30, 2017: Akari Therapeutics Announces FDA Fast Track Designation For Coversin
      • Mar 21, 2017: USPTO Grants Composition of Matter Patent to Achillion for Small Molecule Complement Alternative Pathway Factor D Inhibitors
      • Jan 06, 2017: FDA Grants Orphan Drug Designation To ALXN1210 For The Treatment Of Patients With Paroxysmal Nocturnal Hemoglobinuria
      • Jan 03, 2017: Akari Therapeutics Announces FDA Allowance of IND for Clinical Development of Coversin in PNH
      • Dec 20, 2016: Apellis Announces FDA Fast Track Designation for APL-2 in PNH
      • Dec 05, 2016: Data Presented at American Society of Hematology Meeting Demonstrate Potential Advantages of Factor D Inhibition for the Treatment of Complement Alternative Pathway-Mediated Diseases
      • Dec 05, 2016: Alnylam Presents Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
      • Dec 05, 2016: Akari Therapeutics Announces Corporate Update with New Positive Clinical Data and a New Pipeline of Tick Derived and Engineered Proteins
      • Dec 04, 2016: New Data From ALXN1210 Dose-Escalation Study Presented At ASH Show Rapid And Sustained Reductions In LDH In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
      • Dec 02, 2016: Apellis Reports Positive Interim Results from Phase 1b Clinical Trials of APL-2 in PNH
      • Nov 30, 2016: Ra Pharmaceuticals to Present Overview of RA101495 and Oral C5 Inhibitor at the International PNH Interest Group Annual Scientific Assembly
      • Nov 21, 2016: Apellis to Present Safety and Efficacy Data from Clinical Trials of APL-2 in PNH at December Scientific Meetings
      • Nov 17, 2016: Ra Pharmaceuticals Receives Orphan Drug Designation in the European Union for RA101495 for Treatment of Paroxysmal Nocturnal Hemoglobinuria
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
  • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Companies, H1 2017 (Contd..1), H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Achillion Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Akari Therapeutics Plc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alexion Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amgen Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by F. Hoffmann-La Roche Ltd, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by ISU ABXIS Co Ltd, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Novartis AG, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by NovelMed Therapeutics Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corp, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regeneron Pharmaceuticals Inc, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regenesance BV, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H1 2017
  • Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products, H1 2017

List of Figures

  • Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products by Targets, H1 2017
  • Number of Products by Stage and Targets, H1 2017
  • Number of Products by Mechanism of Actions, H1 2017
  • Number of Products by Stage and Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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