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Paroxysmal Nocturnal Hemoglobinuria Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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    Report

  • 131 Pages
  • June 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5640058
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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Drugs In Development, 2022, 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


The publisher's Pharmaceutical and Healthcare latest pipeline guide Paroxysmal Nocturnal Hemoglobinuria - Drugs In Development, 2022, 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, IND/CTA Filed, Preclinical and Discovery stages are 10, 6, 6, 1, 15 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 the publisher'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

  • Introduction
  • 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
  • Paroxysmal Nocturnal Hemoglobinuria - Drug Profiles
  • Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects
  • Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products
  • Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones
  • Featured News & Press Releases
  • Jun 12, 2022: Samsung Bioepis presents new phase 3 Study of SB12 (Eculizumab), a proposed biosimilar to Soliris, at the European Hematology Association (EHA) Congress 2022
  • Jun 10, 2022: CANbridge CAN106 phase 1 data presented at the European Hematology Association 2022 Congress
  • Jun 10, 2022: Apellis and Sobi report new data reinforcing the robust efficacy and safety profile of EMPAVELI (pegcetacoplan) for PNH at EHA 2022 Congress
  • May 12, 2022: Apellis announces seven abstracts in PNH to be presented at the European Hematology Association Congress
  • May 12, 2022: CANbridge to present CAN106 phase 1 data at the European Hematology Association 2022 Congress
  • Apr 08, 2022: BioCryst pauses enrollment in BCX9930 clinical trials
  • Mar 28, 2022: CANbridge doses first subject in Phase Ib/II PNH treatment trial in China
  • Feb 07, 2022: CANbridge reports positive top-line CAN106 Phase 1 data
  • Feb 04, 2022: Novartis Japan developing Iptacopan as priority product, initially targeting 3 indications
  • Jan 07, 2022: BioCryst begins patient enrollment in REDEEM-1 pivotal trial evaluating BCX9930 as oral monotherapy for patients with PNH
  • Dec 15, 2021: Apellis and Sobi announce EU approval of Aspaveli (pegcetacoplan) for treatment of PNH
  • Dec 13, 2021: Apellis and Sobi Report Empaveli (pegcetacoplan) demonstrated sustained normalization of clinical measures in a broad PNH patient population
  • Dec 11, 2021: Samsung Bioepis’ SB12 Soliris (Eculizumab) Biosimilar Demonstrates PK, PD Bioequivalence in phase 1 study
  • Nov 29, 2021: BioCryst begins patient enrollment in REDEEM-2 pivotal trial evaluating BCX9930 as oral monotherapy for patients with PNH
  • Nov 04, 2021: Apellis to present new data reinforcing EMPAVELI (pegcetacoplan) efficacy and safety in patients with PNH at the 2021 ASH Annual Meeting
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact the Publisher
  • Disclaimer

List of Tables
  • Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, 2022
  • Number of Products under Development by Companies, 2022
  • Products under Development by Companies, 2022
  • Number of Products by Stage and Target, 2022
  • Number of Products by Stage and Mechanism of Action, 2022
  • Number of Products by Stage and Route of Administration, 2022
  • Number of Products by Stage and Molecule Type, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Aevitas Therapeutics Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Akari Therapeutics Plc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alexion Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amgen Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amyndas Pharmaceuticals LLC, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Arrowhead Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Attune Pharmaceuticals, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Biocad, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by BioCryst Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by CANbridge Life Sciences Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Chugai Pharmaceutical Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Homology Medicines Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by ImmunAbs Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by ISU ABXIS Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Kira Pharmaceuticals, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Novartis AG, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by NovelMed Therapeutics Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corp, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Prestige BioPharma Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Rallybio Corp, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Regeneron Pharmaceuticals Inc, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Samsung Bioepis Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Shanghai ComGen Biopharmaceutical Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Shanghai Meiyue Biotechnology Development Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Tasly Biopharmaceuticals Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Turgut Ilaclari AS, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Wuhan LL Science and Technology Development Co Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Zydus Lifesciences Ltd, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, 2022
  • Paroxysmal Nocturnal Hemoglobinuria - Discontinued Products, 2022

List of Figures
  • Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, 2022
  • Number of Products under Development by Companies, 2022
  • Number of Products by Targets, 2022
  • Number of Products by Stage and Targets, 2022
  • Number of Products by Mechanism of Actions, 2022
  • Number of Products by Stage and Mechanism of Actions, 2022
  • Number of Products by Routes of Administration, 2022
  • Number of Products by Stage and Routes of Administration, 2022
  • Number of Products by Top 10 Molecule Types, 2022
  • Number of Products by Stage and Top 10 Molecule Types, 2022

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Aevitas Therapeutics Inc
  • Akari Therapeutics Plc
  • Alexion Pharmaceuticals Inc
  • Alnylam Pharmaceuticals Inc
  • Amgen Inc
  • Amyndas Pharmaceuticals LLC
  • Apellis Pharmaceuticals Inc
  • Arrowhead Pharmaceuticals Inc
  • Attune Pharmaceuticals
  • Biocad
  • BioCryst Pharmaceuticals Inc
  • CANbridge Life Sciences Ltd
  • Chugai Pharmaceutical Co Ltd
  • Homology Medicines Inc
  • ImmunAbs Inc
  • ISU ABXIS Co Ltd
  • Kira Pharmaceuticals
  • Novartis AG
  • NovelMed Therapeutics Inc
  • Omeros Corp
  • Prestige BioPharma Ltd
  • Ra Pharmaceuticals Inc
  • Rallybio Corp
  • Regeneron Pharmaceuticals Inc
  • Samsung Bioepis Co Ltd
  • Shanghai ComGen Biopharmaceutical Co Ltd
  • Shanghai Meiyue Biotechnology Development Co Ltd
  • Tasly Biopharmaceuticals Co Ltd
  • Turgut Ilaclari AS
  • Wuhan LL Science and Technology Development Co Ltd
  • Zydus Lifesciences Ltd