Paroxysmal Nocturnal Hemoglobinuria (PNH) - Drug Pipeline Landscape, 2023

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare disease in which red blood cells break down earlier than normal.

Paroxysmal Nocturnal Hemoglobinuria main causes are age, obesity and hormone therapy, and a genetic disorder.

Paroxysmal Nocturnal Hemoglobinuria symptoms includes abdominal pain, back pain, dark urine, easy bruising or bleeding, headache, and shortness of breath.

Paroxysmal Nocturnal Hemoglobinuria is diagnosed by tests like flow cytometry, a blood test that identifies blood cells missing GPI-anchored proteins.

Paroxysmal nocturnal hemoglobinuria treatment includes surgery, chemotherapy, radiation therapy, allogeneic bone marrow transplantation, and drugs like terminal complement activation inhibitors.

Report Highlights

Global Insight Service’s, Paroxysmal Nocturnal Hemoglobinuria (PNH) - Drug Pipeline Landscape, 2023 report provides an overview of the Paroxysmal Nocturnal Hemoglobinuria (PNH) pipeline drugs. This report covers detailed insights on Paroxysmal Nocturnal Hemoglobinuria (PNH) drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Paroxysmal Nocturnal Hemoglobinuria (PNH) pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.

Methodology

The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.

Scope

The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.

Reasons to Buy

Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.

Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.

Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.

Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

Please Note: This report will be delivered by publisher within 3-4 business days of order confirmation.


List of Tables
List of Figures
1. Introduction
1.1 Paroxysmal Nocturnal Hemoglobinuria (PNH) – Pipeline Drugs, 2023 - Coverage
2. Disease Overview – Paroxysmal Nocturnal Hemoglobinuria (PNH)
2.1 Causes
2.2 Signs and Symptoms
2.3 Diagnosis
2.4 Disease Management
3. Paroxysmal Nocturnal Hemoglobinuria (PNH) – Pipeline Drugs Development - Overview
3.1 Comparative Analysis by Stage of Development
3.2 Number of Products under Development by Companies, 2023
3.3 Products under Development by Companies, 2023
4. Assessment of Therapeutics
4.1 Assessment by Target
4.2 Assessment by Mechanism of Action
4.3 Assessment by Molecule Type
4.3 Assessment by Route of Administration
5. Drug Profiles
5.1 Clinical Stage Drugs – Pre-Registration
5.1.1 RG6107
5.1.2 SB12
5.2 Clinical Stage Drugs – Phase III
5.2.1 ABP 959
5.2.2 ALXN2040
5.2.3 LNP023
5.2.4 Pozelimab + Cemdisira
5.2.5 Ultomiris
5.3 Clinical Stage Drugs – Phase II
5.3.1 ALXN2050
5.3.2 ARO-C3
5.3.3 CAN 106
5.3.4 KP104
5.3.5 LFG316
5.4 Clinical Stage Drugs – Phase I
5.4.1 AMY-101
5.4.2 IM-101
5.4.3 ISU305
5.4.4 MY008211A
5.4.5 NM8074
5.4.6 OMS906
5.4.7 RLYB-116
5.5 Clinical Stage Drugs – Preclinical
5.5.1 CG001
5.5.2 HMI-104
5.5.3 NM3086
5.5.4 QR059
5.6 Clinical Stage Drugs – Discovery
5.6.1 PBP1603
6. Key Regulatory Designations
7. Key Deals
8. Key Upcoming Milestones
9. Key Companies Involved
9.1 Alexion Pharmaceuticals
9.2 Amgen
9.3 Amyndas Pharmaceuticals S.A
9.4 Arrowhead Pharmaceuticals
9.5 CANbridge Life Sciences Ltd
9.6 Hoffmann-La Roche
9.7 Homology Medicines Inc
9.8 ImmunAbs Inc
9.9 ISU ABXIS Co Ltd
9.10 Kira Pharmacenticals LLC
9.11 Novartis Pharmaceuticals
9.12 Novel Med Therapeutics
9.13 Omeros Corp
9.14 Prestige BioPharma Ltd
9.15 Rallybio Corp
9.16 Regeneron Pharmaceuticals
9.17 Samsung Bioepis Co Ltd
9.18 Shanghai ComGen Biopharmaceutical Co Ltd
9.19 Wuhan Createrna Science and Technology CO Ltd
10. Dormant Drugs
10.1 Inactive Drugs
10.2 Discontinued Drugs
11. Appendix
List of Tables
Table 1.1 Number of Products Under Development for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Table 1.2 Number of Products Under Development by Companies
Table 1.3 Products Under Development by Companies
Table 1.4 Products by Targets
Table 1.5 Products by Mechanism of Action
Table 1.6 Products by Molecule Type
Table 1.7 Products by Route of Administration
Table 2.1 Clinical Trial Details - ABP 959/Amgen
Table 2.2 Clinical Trial Details - ALXN2040/Alexion Pharmaceuticals
Table 2.3 Clinical Trial Details - LNP023/Novartis Pharmaceuticals
Table 2.4 Clinical Trial Details - Pozelimab + Cemdisiran/Regeneron Pharmaceuticals
Table 2.5 Clinical Trial Details – Ultomiris (Pediatric)/Alexion Pharmaceuticals
Table 2.6 Clinical Trial Details - ALXN2050/Alexion Pharmaceuticals
Table 2.7 Clinical Trial Details - ARO-C3/Arrowhead Pharmaceuticals
Table 2.8 Clinical Trial Details - CAN 106/CANbridge Life Sciences Ltd
Table 2.9 Clinical Trial Details - KP104/Kira Pharmacenticals LLC
Table 2.10 Clinical Trial Details - LFG316/Novartis Pharmaceuticals
Table 2.11 Clinical Trial Details - AMY-101/Amyndas Pharmaceuticals S.A
Table 2.12 Clinical Trial Details - ISU305/ISU ABXIS Co Ltd
Table 2.13 Clinical Trial Details - MY008211A/Wuhan Createrna Science and Technology Co Ltd
Table 2.14 Clinical Trial Details - NM8074/Novel Med Therapeutics
Table 3.1 Regulatory Designations
Table 4.1 Inactive Drugs
Table 4.2 Discontinued Drugs
List of Figures
Figure 1.1 Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria (PNH), 2023
Figure 1.2 Products by Top 5 Targets and Stage of Development for Paroxysmal Nocturnal Hemoglobinuria (PNH), 2023
Figure 1.3 Products by Top 5 Mechanism of Action for Paroxysmal Nocturnal Hemoglobinuria (PNH), 2023
Figure 1.4 Products by Top 5 Molecule Type for Paroxysmal Nocturnal Hemoglobinuria (PNH), 2023
Figure 1.5 Products by Top 5 Route of Administration for Paroxysmal Nocturnal Hemoglobinuria (PNH), 2023

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