Hemophilia A Pipeline Drugs Analysis Report, 2024 Updates: FDA Approvals, Clinical Trials, Therapies, MOA, ROA by DelveInsight | Genentech, Novo Nordisk, Pfizer, Sanofi, Shire, ApcinteX, CSL Behring

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 Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Hemophilia A Market.

Some of the key takeaways from the Hemophilia A Pipeline Report: 

• Companies across the globe are diligently working toward developing novel Hemophilia A treatment therapies with a considerable amount of success over the years. 
• Hemophilia A companies working in the treatment market are Generation Bio, Poseida Therapeutics, Expression Therapeutics, Chugai Pharmaceutical, ASC Therapeutics, Ascension, Staidson (Beijing) Biopharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, Novo Nordisk, Pfizer, and others, are developing therapies for the Hemophilia A treatment 
• Emerging Hemophilia A therapies in the different phases of clinical trials are- Research program: Gene Therapies, P-FVIII-101, ET3 lentiviral gene therapy, NXT007, ASC618, SelectAte, STSP-0601, TQG 203, Mim8, Giroctocogene fitelparvovec, and others are expected to have a significant impact on the Hemophilia A market in the coming years.
• In December 2023, Roche has released preliminary analysis findings from the Phase III HAVEN 7 clinical trial, demonstrating the efficacy of Hemlibra (emicizumab), a bispecific antibody targeting factor IXa and factor X, in infants suffering from severe hemophilia A but lacking factor VIII inhibitors.
• In January 2023: worldwide biotechnology business BioMarin Pharmaceutical Inc., committed to improving lives through genetic research, released encouraging findings from its ongoing worldwide Phase 3 GENEr8-1 study of ROCTAVIANTM (valoctocogene roxaparvovec) after more than three years of follow-up. An experimental one-time gene therapy is being used to treat adults with severe hemophilia A. With 134 participants, it is the largest and most extensive global Phase 3 study for any gene treatment in hemophilia.In February 2023, For adults and children with hemophilia A, the US FDA approved ALTUVIIIO (efanesoctocog alfa) as a factor VIII replacement medication. For adults and children with hemophilia A, ALTUVIIIO is recommended for perioperative management (surgery), routine prophylaxis, and on-demand medication to control bleeding episodes.
• In September 2022, Reopening recruitment for the Phase III AFFINE trial, which is assessing giroctocogene fitelparvovec, is a joint announcement from Pfizer and Sangamo Therapeutics.
• In August 2022, For the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5, the European Commission (EC) granted conditional marketing permission to ROCTAVIAN gene therapy.

Hemophilia A Overview

A genetic bleeding illness called hemophilia A, or classical hemophilia, is brought on by low amounts of a blood protein called factor VIII. Hemophilia A patients will bleed more profusely following an injury, procedure, or surgery.

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Emerging Hemophilia A Drugs Under Different Phases of Clinical Development Include:

• Research program: Gene Therapies: Generation Bio
• P-FVIII-101: Poseida Therapeutics
• ET3 lentiviral gene therapy: Expression Therapeutics
• NXT007: Chugai Pharmaceutical
• ASC618: ASC Therapeutics
• SelectAte: Ascension
• STSP-0601: Staidson (Beijing) Biopharmaceutic als
• TQG 203: Chia Tai Tianqing Pharmaceutical Group
• Mim8: Novo Nordisk
• Giroctocogene fitelparvovec: Pfizer

Hemophilia A 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
• Intravenous
• Subcutaneous
• Topical

Hemophilia A Molecule Type

Hemophilia A Products have been categorized under various Molecule types, such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy 

Hemophilia A Pipeline Therapeutics Assessment

• Hemophilia A Assessment by Product Type
• Hemophilia A By Stage and Product Type
• Hemophilia A Assessment by Route of Administration
• Hemophilia A By Stage and Route of Administration
• Hemophilia A Assessment by Molecule Type
• Hemophilia A by Stage and Molecule Type

DelveInsight’s Hemophilia A Report covers around 45+ 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

Further Hemophilia A product details are provided in the report. Download the Hemophilia A pipeline report to learn more about the emerging Hemophilia A therapies

Some of the key companies in the Hemophilia A Therapeutics Market include:

Key companies developing therapies for Hemophilia A are – ApcinteX, ASC Therapeutics, Ultragenix Pharmaceutical, BioMarin Pharmaceutical, CSL Behring, Freeline Therapeutics, Genentech, Inc., Novo Nordisk, Pfizer, Sanofi, Shire, Spark Therapeutics, Amarna therapeutics, Asklepios BioPharmaceutical, Bayer, Belief Biomed, Bioverativ, Catalyst Biosciences, Centessa Pharmaceuticals, Chameleon Biosciences, Chia Tai Tianqing Pharmaceutical Group, Expression Therapeutics, GC Pharma, GeneVentiv, Intellia tx, OPKO Health, Sangamo Therapeutics, Staidson Beijing BioPharmaceuticals, UBI Pharma, uniQure, and others.

Hemophilia A Pipeline Analysis:

The Hemophilia A pipeline report provides insights into 

• The report provides detailed insights about companies that are developing therapies for the treatment of Hemophilia A with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia A Treatment.
• Hemophilia A key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Hemophilia A Drugs 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. 
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of 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.

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Hemophilia A Pipeline Market Drivers

• Increasing number of hemophilic patients worldwide, increasing development of novel therapies, increasing diagnostic rate are some of the important factors that are fueling the Hemophilia A Market.

Hemophilia A Pipeline Market Barriers

• However, side effects associated with the plasma derived products, high cost of recombinant products and other factors are creating obstacles in the Hemophilia A Market growth.

Scope of Hemophilia A Pipeline Drug Insight    

• Coverage: Global
• Key Hemophilia A Companies: Generation Bio, Poseida Therapeutics, Expression Therapeutics, Chugai Pharmaceutical, ASC Therapeutics, Ascension, Staidson (Beijing) Biopharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, Novo Nordisk, Pfizer, and others
• Key Hemophilia A Therapies: Research program: Gene Therapies, P-FVIII-101, ET3 lentiviral gene therapy, NXT007, ASC618, SelectAte, STSP-0601, TQG 203, Mim8, Giroctocogene fitelparvovec, and others
• Hemophilia A Therapeutic Assessment: Hemophilia A current marketed and Hemophilia A emerging therapies
• Hemophilia A Market Dynamics: Hemophilia A market drivers and Hemophilia A market barriers 

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Table of Contents

1. Hemophilia A Report Introduction

2. Hemophilia A Executive Summary

3. Hemophilia A Overview

4. Hemophilia A- Analytical Perspective In-depth Commercial Assessment

5. Hemophilia A Pipeline Therapeutics

6. Hemophilia A Late Stage Products (Phase II/III)

7. Hemophilia A Mid Stage Products (Phase II)

8. Hemophilia A Early Stage Products (Phase I)

9. Hemophilia A Preclinical Stage Products

10. Hemophilia A Therapeutics Assessment

11. Hemophilia A Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Hemophilia A Key Companies

14. Hemophilia A Key Products

15. Hemophilia A Unmet Needs

16 . Hemophilia A Market Drivers and Barriers

17. Hemophilia A Future Perspectives and Conclusion

18. Hemophilia A Analyst Views

19. Appendix

20. About DelveInsight

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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