DelveInsight’s ‘Hemophilia A Pipeline Insight 2022‘ 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.
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, Research program: gene therapies, GENV-HEM, Research program: gene editing therapeutics, NX-01, and others.
- In November 2022, CSL Limited announced that its subsidiary, CSL Seqirus, has entered into a collaboration and license agreement with Arcturus Therapeutics Holdings Inc (“Arcturus Therapeutics”) to gain access to their late-stage self-amplifying mRNA (sa-mRNA) vaccine platform technology.
- In October 2022, the Food and Drug Administration accepted BioMarin Pharmaceutical’s latest approval application for a gene therapy designed to treat severe hemophilia A, and a decision date of March 31 has been set.
Request a sample and discover the recent breakthroughs happening Hemophilia A pipeline landscape in @Hemophilia A Pipeline Report
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 Analysis: Drug Profile
AGN-193408: Allergan
Allergan is evaluating the safety and efficacy of AGN-193408 SR in participants with open-angle glaucoma or ocular hypertension in a multicenter, open-label, dose-escalation (Cohort 1) to masked, randomized, parallel-group (Cohort 2) study. It is a biodegradable polymer matrix implant containing preservative-free AGN-193408.
OCTA101: Octapharma
Octapharma is testing OCTA101 (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer) in a Phase 1/2 study in adults in 5 cohorts, with the primary goal of determining the safety of subcutaneous injection of OCTA101 (in previously treated adult patients with severe hemophilia A). The study also intends to compare the PK characteristics, dose proportionality, and subcutaneous bioavailability of OCTA101 to intravenous administration of Nuwiq (Human-cl RH FVIII) to define the prophylactic treatment (dose and injection interval) that would result in protective trough levels of FVIII: C for future Phase 3 studies.
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
Learn more about the Hemophilia A emerging pipeline therapies @Hemophilia A Clinical Trials
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
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, and others.
Dive deep into rich insights for Hemophilia A emerging therapies and assessment; visit @Hemophilia A Emerging Therapies
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Overview |
| 4. | 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. | Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Collaborations Assessment- Licensing / Partnering / Funding |
| 13. | Unmet Needs |
| 14. | Market Drivers and Barriers |
| 15. | Appendix |
| 16. | About DelveInsight |
For further information on the Hemophilia A current pipeline therapeutics, reach out @Hemophilia A Therapeutics Assessment
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