Hemophilia A Pipeline Assessment, 2023 Updates | In-depth Insights into the Clinical and Non-Clinical Trials, Emerging Drugs, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, and Key Companies

February 28 03:29 2023
Hemophilia A Pipeline Assessment, 2023 Updates | In-depth Insights into the Clinical and Non-Clinical Trials, Emerging Drugs, Latest FDA, EMA, and PMDA Approvals, Treatment Outlook, and Key Companies
Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, about 35+ key pharma and biotech companies are working on 45+ pipeline drugs in the Hemophilia A therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Currently, the major treatment options for Hemophilia A are Factor Replacement Concentrates, the source of which is either recombinant DNA technology or Human plasma-derive, and Bypassing agents. Moreover, this is an off-label treatment option, wherein Desmopressin Acetate (DDAVP) and Adjunctive therapies are also available for the management of Hemophilia A.

Companies like BioMarin Pharmaceutical, Novo Nordisk, Sanofi, Pfizer, Sangamo Therapeutics, Spark Therapeutics, and others are coming up with novel therapeutic approaches that can entirely change the treatment landscape of Hemophilia A. If launched, these emerging potential candidates could pose an adverse threat to current hemophilia A pharmaceutical giants by means of their potential to transform the standard of care for hemophilia A patients.

Hemophilia A Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Hemophilia A Market. 

The Hemophilia A Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Hemophilia A Pipeline Analysis

Hemophilia A Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the emerging therapies for the treatment of Hemophilia A and the aggregate therapies developed by major pharma companies.

  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Hemophilia A market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.  

Analysis of Emerging Therapies by Phases

The report covers the emerging products under different phases of clinical development like –

  • Late stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage product (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates


Route of Administration

Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as 

  • Oral

  • Parenteral

  • Intravitreal 

  • Subretinal

  • Topical


Molecule Type

Products have been categorized under various Molecule types, such as

  • Monoclonal Antibody

  • Peptides 

  • Polymer 

  • Small molecule

  • Gene therapy


Mechanism of Action of the Emerging Pipeline Therapies

  • Factor VIII replacements

  • Blood coagulation factor stimulants

  • Factor X stimulants

  • Factor VIIa stimulants

  • Factor VIII replacements

  • Gene transference

Learn How the Ongoing Clinical & Commercial Activities will Affect the Hemophilia A Therapeutic Segment @



Hemophilia A Therapeutics Landscape

The Hemophilia A therapeutic market dynamic is anticipated to evolve immensely in the coming years owing to the rise in the prevalent population of Hemophilia A, the expected entry of premium price assets such as gene therapy, siRNA, and bispecific antibodies, and the expected readily uptake of recently approved therapies. 

There are approx. 35+ key companies developing therapies for Hemophilia A. Currently, Novo Nordisk is leading the therapeutics market with its Hemophilia A drug candidates in the most advanced stage of clinical development.

The Leading Companies in the Hemophilia A Therapeutics Market Include:

Alnylam Pharmaceuticals, ASC Therapeutics, Ascension, BioMarin Pharmaceutical, Catalyst Biosciences, Centessa Pharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, Chugai Pharmaceuticals, Dimension Therapeutics, Expression Therapeutics, Hoffmann-La Roche, Intellia Therapeutics, Kymab, Novo Nordisk, Pfizer, Poseida Therapeutics, Regeneron Pharmaceuticals, Sanofi Genzyme, Spark Therapeutics, Staidson Beijing BioPharmaceuticals, and many others.

Hemophilia A Drugs Covered in the Report Include:

  • ASC618: ASC Therapeutics

  • BIVV001: Sanofi/Sobi

  • Concizumab: Novo Nordisk

  • ET3 lentiviral gene therapy: Expression Therapeutics

  • Fitusiran: Sanofi/Alnylam Pharmaceuticals

  • Giroctocogene fitelparvovec (SB-525/PF-07055480): Pfizer/Sangamo Therapeutics

  • Marstacimab: Pfizer

  • Marzeptacog alfa: Catalyst Biosciences

  • Mim8: Nono Nordisk

  • NXT007: Chugai Pharmaceuticals

  • PF-07055480: Pfizer

  • RG6357 (SPK-8011), RG6358 (SPK-8016): Roche/Spark Therapeutics

  • SelectAte: Ascension

  • STSP-0601: Staidson (Beijing) Biopharmaceuticals

  • TQG 203: Chia Tai Tianqing

  • Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical

  • Valrox: BioMarin Pharmaceutical

And Many More

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @


Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Hemophilia A Current Treatment Patterns

4. Hemophilia A – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Hemophilia A Late Stage Products (Phase-III)

7. Hemophilia A Mid-Stage Products (Phase-II)

8. Hemophilia A Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Hemophilia A Discontinued Products

13. Hemophilia A Product Profiles

14. Key Companies in the Hemophilia A Market

15. Key Products in the Hemophilia A Therapeutics Segment

16. Dormant and Discontinued Products

17. Hemophilia A Unmet Needs

18. Hemophilia A Future Perspectives

19. Hemophilia A Analyst Review  

20. Appendix

21. Report Methodology

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download Sample PDF to Explore the Key Offerings of the Report @



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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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