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

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Published: June 30, 2022 Report Code: GDGMDHC22208IDB-ST

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The symptoms of Paroxysmal Nocturnal Hemoglobinuria include abdominal pain, back pain, dark urine, easy bruising or bleeding, headache, and shortness of breath. Its treatment includes surgery, chemotherapy, and radiation therapy.

Key Targets Complement C5, Complement Factor D, Complement C3, Leukotriene B4, Complement Factor B, Mannan Binding Lectin Serine Protease 1, and Properdin
Key Mechanisms of Action Complement C5 Inhibitor, Complement Factor D Inhibitor, Complement C3 Inhibitor, Leukotriene B4 Inhibitor, Complement Factor B Inhibitor, Mannan Binding Lectin Serine Protease 1 Inhibitor, and Properdin Inhibitor
Key Routes of Administration Intravenous, Subcutaneous, Oral, Intravitreal, Inhalational, Ophthalmic, and Intravenous Drip
Key Molecule Type Monoclonal Antibody, Small Molecule, Antisense RNAi Oligonucleotide, Biologic, Enzyme, Fusion Protein, Gene Therapy, Recombinant Protein, Synthetic Peptide, and Peptide
Major Companies Alexion Pharmaceuticals Inc, NovelMed Therapeutics Inc, Akari Therapeutics Plc, Alnylam Pharmaceuticals Inc, Apellis Pharmaceuticals Inc, CANbridge Life Sciences Ltd, Novartis AG, Regeneron Pharmaceuticals Inc, Wuhan LL Science and Technology Development Co Ltd, and Aevitas Therapeutics Inc

The Paroxysmal Nocturnal Hemoglobinuria pipeline market research report provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and features dormant and discontinued projects.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market - Key Targets

The key targets in the Paroxysmal Nocturnal Hemoglobinuria pipeline products market are Complement C5, Complement Factor D, Complement C3, Leukotriene B4, Complement Factor B, Mannan Binding Lectin Serine Protease 1, and Properdin.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Targets

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Targets

For more target insights, download a free report sample

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market - Key Mechanisms of Action

The key mechanisms of action in the Paroxysmal Nocturnal Hemoglobinuria pipeline products market are Complement C5 Inhibitor, Complement Factor D Inhibitor, Complement C3 Inhibitor, Leukotriene B4 Inhibitor, Complement Factor B Inhibitor, Mannan Binding Lectin Serine Protease 1 Inhibitor, and Properdin Inhibitor.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Mechanisms of Action

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Mechanisms of Action

For more mechanisms of action insights, download a free report sample

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market - Key Routes of Administration

The key routes of administration in the Paroxysmal Nocturnal Hemoglobinuria pipeline products market are intravenous, subcutaneous, oral, intravitreal, inhalational, ophthalmic, and intravenous drip.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Routes of Administration

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Routes of Administration

For more routes of administration insights, download a free report sample

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market - Key Molecule Types

The key molecule types in the Paroxysmal Nocturnal Hemoglobinuria pipeline products market are monoclonal antibody, small molecule, antisense RNAi oligonucleotide, biologic, enzyme, fusion protein, gene therapy, recombinant protein, synthetic peptide, and peptide.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Molecule Types

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Molecule Types

For more molecule type insights, download a free report sample

Competitive Landscape

Some of the major companies in the Paroxysmal Nocturnal Hemoglobinuria pipeline products market are Alexion Pharmaceuticals Inc, NovelMed Therapeutics Inc, Akari Therapeutics Plc, Alnylam Pharmaceuticals Inc, Apellis Pharmaceuticals Inc, CANbridge Life Sciences Ltd, Novartis AG, Regeneron Pharmaceuticals Inc, Wuhan LL Science and Technology Development Co Ltd, and Aevitas Therapeutics Inc.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Companies

Paroxysmal Nocturnal Hemoglobinuria Pipeline Products Market Analysis by Companies

To know more about major companies, download a free report sample

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 the 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 portfolios 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 their 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 the pipeline.

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

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

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 Us

Disclaimer

Table

List of Tables

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Companies, 2022 (Contd..1)

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Products under Development by Companies, 2022 (Contd..2)

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

Figures

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

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