(Albany, USA) DelveInsight’s ‘Hemophilia A Pipeline Insight 2024‘ report provides comprehensive global coverage of available, marketed, and pipeline Hemophilia A therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Hemophilia A pipeline domain.
For Hemophilia A emerging drugs, the Hemophilia A pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
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Key Takeaways from the Hemophilia A Pipeline Report
- Over 40+ Hemophilia A pipeline therapies are in various stages of development, and their anticipated acceptance in the Hemophilia A market would significantly increase market revenue.
- Leading Hemophilia A companies developing novel drug candidates to improve the Hemophilia A treatment landscape include Spark Therapeutics, Sigilon Therapeutics, ASC Therapeutics, Pfizer, Sanofi Genzyme, Novo Nordisk, Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd, Bayer, Ultragenix pharmaceutical, ApcinteX Ltd, Chia Tai Tianqing Pharmaceutical Group Co., Ltd, Expression Therapeutics, LLC, CSL Behring, ASC Therapeutics, Poseida Therapeutics, Staidson Beijing BioPharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, GeneVentiv Therapeutics, Jiangsu Gensciences, 2seventy bio, Generation Bio, Apitope Technology, and others.
- Promising Hemophilia A pipeline therapies in various stages of development include SPK-8011, SPK-8016, BIVV001, AGN-193408, Valoctocogene roxaparvovec, P-FVIII-101, STSP 0601, TQG203, SIG-001, Mim8, PF-07055480, Efanesoctocog alfa, WP 1301, Advate, Recombinate, antihemophilic factor, Eloctate, Hemlibra, Cyklokapron, NovoSeven RT, Xyntha, Humate-P, Hemofil-M, Adynovate, Afstyla, Feiba VH, Kogenate FS, Alphanate, Helixate FS, Autoplex T, Jivi, Nuwiq, Research program: gene therapies, GENV-HEM, Research program: gene editing therapeutics, NX-01, and others.
- In June of 2023, BioMarin disclosed that the FDA had granted approval to Roctavian for use as a single-dose therapy in individuals with severe hemophilia A.
- In February 2023, the FDA in the United States gave the green light to ALTUVIIIO (efanesoctocog alfa), a replacement therapy for factor VIII, for both adults and children diagnosed with hemophilia A. ALTUVIIIO is prescribed for regular preventive measures, as well as for addressing bleeding incidents when needed, and for managing the perioperative period (surgery) in adults and children suffering from hemophilia A.
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Hemophilia A Overview
Hemophilia is an inherited rare bleeding disorder in which blood does not clot properly because the affected person does not produce enough blood-clotting proteins (clotting factors). Patients who are injured cannot stop bleeding unless these factors are present. The severity of hemophilia symptoms is determined by the level of clotting factors. If the clotting factor level is mildly reduced, the patient may only bleed after surgery or trauma. If the deficiency is severe, one can easily bleed for no apparent reason. Small cuts are not dangerous, but internal bleeding is extremely dangerous for Hemophilia patients. Internal bleeding in the elbows, knees, ankles and other joints is the main concern with this genetic disorder. Internal bleeding can cause organ and tissue damage as well as be potentially fatal.
Hemophilia is classified into several types, including Hemophilia A, Hemophilia B, Hemophilia C, and Von Willebrand disease. Hemophilia is classified based on the presence of clotting factors, such as factor VIII in Hemophilia A and factor IX in Hemophilia B. Screening tests for hemophilia include the Complete Blood Count (CBC), Activated Partial Thromboplastin Time (APTT) Test, Prothrombin Time (PT) Test, Fibrinogen Test, and clotting factor tests.
Hemophilia A Pipeline Therapies and Key Companies
- Efanesoctocog alfa: Sanofi
- FRSW 107: Jiangsu Gensciences
- Mim8: Novo Nordisk
- SPK-8016: Spark Therapeutics
- OCTA101: Octapharma
- FRSW 117: Jiangsu Gensciences
- TQG203: Chia Tai Tianqing/Pharmaceutical Group
- STSP 0601: Staidson Beijing/BioPharmaceuticals
- P-FVIII-101: Poseida Therapeutics
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Hemophilia A Pipeline Therapeutics Assessment
By Product Type
- Monotherapy
- Combination Therapy
By Stage
- Discovery
- Pre-Clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
By Route of Administration
- Oral
- Intravenous
- Subcutaneous
By Molecule Type
- Small molecules
- Gene Therapies
- Bispecific antibodies
- Recombinant proteins
- Fusion Proteins
- Coagulants
- Blood coagulation factor replacements
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Scope of the Hemophilia A Pipeline Report
- Coverage: Global
- Key Hemophilia A Companies: Spark Therapeutics, Sigilon Therapeutics, ASC Therapeutics, Pfizer, Sanofi Genzyme, Novo Nordisk, Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd, Bayer, Ultragenix pharmaceutical, ApcinteX Ltd, Chia Tai Tianqing Pharmaceutical Group Co., Ltd, Expression Therapeutics, LLC, CSL Behring, ASC Therapeutics, Poseida Therapeutics, Staidson Beijing BioPharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, GeneVentiv Therapeutics, Jiangsu Gensciences, 2seventy bio, Generation Bio, Apitope Technology, and others.
- Key Hemophilia A Pipeline Therapies: SPK-8011, SPK-8016, BIVV001, AGN-193408, Valoctocogene roxaparvovec, P-FVIII-101, STSP 0601, TQG203, SIG-001, Mim8, PF-07055480, Efanesoctocog alfa, WP 1301, Research program: gene therapies, GENV-HEM, Research program: gene editing therapeutics, NX-01, Advate, Recombinate, antihemophilic factor, Eloctate, Hemlibra, Cyklokapron, NovoSeven RT, Xyntha, Humate-P, Hemofil-M, Adynovate, Afstyla, Feiba VH, Kogenate FS, Alphanate, Helixate FS, Autoplex T, Jivi, Nuwiq and others.
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Table of Contents
1. Introduction
2. Executive Summary
3. Hemophilia A Overview
4. Hemophilia A Pipeline Therapeutics
5. Late Stage Products (Phase III)
6. Mid Stage Products (Phase II)
7. Early Stage Products (Phase I/II)
8. Preclinical Stage Products
9. Discovery Stage Products
10. Hemophilia A Therapeutic Assessment
11. Inactive Hemophilia A Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Hemophilia A Unmet Needs
14. Hemophilia A Market Drivers and Barriers
15. Appendix
16. About DelveInsight
About DelveInsight
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