The Hemophilia 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 Pipeline Insight, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Hemophilia Market.
Some of the key takeaways from the Hemophilia Pipeline Report:
- Companies across the globe are diligently working toward developing novel Hemophilia treatment therapies with a considerable amount of success over the years.
- Hemophilia companies working in the treatment market are Poseida Therapeutics, GC Biopharma Corp, ASC Therapeutics, Centessa Pharmaceuticals plc, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, Pfizer, Nordisk A/S, uniQure, Alnylam Pharmaceuticals, Freeline Therapeutics, ASC Therapeutics, GeneVentiv, and others, are developing therapies for the Hemophilia treatment
- Emerging Hemophilia therapies in the different phases of clinical trials are- P-FVIII-101, MG1113, ASC618, SerpinPC, STSP-0601, SPK-8011, PF-07055480, Mim8Novo, Etranacogene dezaparvovec, Fitusiran, FLT-180a, ASC 618, GENV-001, and others are expected to have a significant impact on the Hemophilia market in the coming years.
- 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 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 longest and biggest Phase 3 study ever conducted globally for any kind of gene therapy for hemophilia.
- In September 2022, The Phase III AFFINE study, which is assessing giroctocogene fitelparvovec as an experimental gene therapy for individuals with hemophilia A ranging from moderately severe to severe, has reopened enrollment, according to a statement from Pfizer and Sangamo Therapeutics. By the end of 2022, all trial sites should be operational, and in the first half of 2024, a crucial readout is anticipated.
- In June 2022, The second cohort in Freeline Therapeutics Holdings’ Phase I/II B-LIEVE dose confirmation trial of FLT180a in hemophilia B patients is already being dosed.
- In May 2022, UniQure declared that the FDA had accepted the Biologics License Application (BLA) for etranacogene dezaparvovec for priority review. Drugs that, if approved, could potentially significantly improve the safety or efficacy of the treatment, diagnosis, or prevention of serious illnesses relative to standard applications are designated for priority review of a BLA.
- In March 2022, ASC618 was given Fast Track designation by the Food and Drug Administration (FDA) to treat hemophilia A.
- In January 2022, In order to collaboratively investigate and develop an in vivo gene editing treatment for hemophilia A, 2seventy bio announced that it has entered into an option and license agreement with Novo Nordisk. The two companies have a successful multi-year research collaboration that is built upon with this arrangement.
Hemophilia is an uncommon bleeding condition characterized by improper blood clotting. This may result in excessive bleeding issues following surgery or an injury.
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Emerging Hemophilia Drugs Under Different Phases of Clinical Development Include:
- P-FVIII-101: Poseida Therapeutics
- MG1113: GC Biopharma Corp
- ASC618: ASC Therapeutics
- SerpinPC: Centessa Pharmaceuticals plc
- STSP-0601: Staidson Beijing BioPharmaceuticals
- SPK-8011: Spark Therapeutics
- PF-07055480: Pfizer
- Mim8Novo: Nordisk A/S
- Etranacogene dezaparvovec: uniQure
- Fitusiran: Alnylam Pharmaceuticals
- FLT-180a: Freeline Therapeutics
- ASC 618: ASC Therapeutics
- GENV-001: GeneVentiv
Hemophilia Route of Administration
Hemophilia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
Hemophilia Molecule Type
Hemophilia Products have been categorized under various Molecule types, such as
- Small molecule
Hemophilia Pipeline Therapeutics Assessment
- Hemophilia Assessment by Product Type
- Hemophilia By Stage and Product Type
- Hemophilia Assessment by Route of Administration
- Hemophilia By Stage and Route of Administration
- Hemophilia Assessment by Molecule Type
- Hemophilia by Stage and Molecule Type
DelveInsight’s Hemophilia Report covers around 80+ 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 product details are provided in the report. Download the Hemophilia pipeline report to learn more about the emerging Hemophilia therapies
Some of the key companies in the Hemophilia Therapeutics Market include:
Key companies developing therapies for Hemophilia 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, and others.
Hemophilia Pipeline Analysis:
The Hemophilia pipeline report provides insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Hemophilia 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 Treatment.
- Hemophilia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Hemophilia 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 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 Pipeline Market Drivers
- Growing burden on regulatory bodies towards Hemophilia treatment is resulted to increase R&D efforts, advancements in rare disease gene therapy market coupled with increasing research and development activities will favor the growth of hemophilia treatment market are some of the important factors that are fueling the Hemophilia Market.
Hemophilia Pipeline Market Barriers
- However, possible market access issues for gene therapies due to their high upfront costs, lack of long-term data, and robust evidence of efficacy and safety, high cost of the treatment and other factors are creating obstacles in the Hemophilia Market growth.
Scope of Hemophilia Pipeline Drug Insight
- Coverage: Global
- Key Hemophilia Companies: Poseida Therapeutics, GC Biopharma Corp, ASC Therapeutics, Centessa Pharmaceuticals plc, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, Pfizer, Nordisk A/S, uniQure, Alnylam Pharmaceuticals, Freeline Therapeutics, ASC Therapeutics, GeneVentiv, and others
- Key Hemophilia Therapies: P-FVIII-101, MG1113, ASC618, SerpinPC, STSP-0601, SPK-8011, PF-07055480, Mim8Novo, Etranacogene dezaparvovec, Fitusiran, FLT-180a, ASC 618, GENV-001, and others
- Hemophilia Therapeutic Assessment: Hemophilia current marketed and Hemophilia emerging therapies
- Hemophilia Market Dynamics: Hemophilia market drivers and Hemophilia market barriers
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Table of Contents
1. Hemophilia Report Introduction
2. Hemophilia Executive Summary
3. Hemophilia Overview
4. Hemophilia- Analytical Perspective In-depth Commercial Assessment
5. Hemophilia Pipeline Therapeutics
6. Hemophilia Late Stage Products (Phase II/III)
7. Hemophilia Mid Stage Products (Phase II)
8. Hemophilia Early Stage Products (Phase I)
9. Hemophilia Preclinical Stage Products
10. Hemophilia Therapeutics Assessment
11. Hemophilia Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Hemophilia Key Companies
14. Hemophilia Key Products
15. Hemophilia Unmet Needs
16 . Hemophilia Market Drivers and Barriers
17. Hemophilia Future Perspectives and Conclusion
18. Hemophilia Analyst Views
20. 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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