Hemophilia A Pipeline Analysis Demonstrates Novel 35+ Therapies at the Horizon Expected to Transform the Treatment Paradigm | DelveInsight

Hemophilia A Pipeline Analysis Demonstrates Novel 35+ Therapies at the Horizon Expected to Transform the Treatment Paradigm | DelveInsight

DelveInsight’s, “Hemophilia A Pipeline Insight, 2023,” report provides comprehensive insights about 35+ companies and 45+ pipeline drugs in Hemophilia A pipeline landscape. It covers the Hemophilia A pipeline drug profiles, including Hemophilia A clinical trials and nonclinical stage products. It also covers the Hemophilia A pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

In the Hemophilia A Pipeline Report, detailed description of the drug is given which includes mechanism of action of the drug, Hemophilia A clinical trials studies, Hemophilia A NDA approvals (if any), and product development activities comprising the technology, Hemophilia A collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Key takeaways from the Hemophilia A Pipeline Report

 

  • DelveInsight’s Hemophilia A Pipeline report depicts a robust space with 35+ active players working to develop 45+ pipeline therapies for Hemophilia A treatment.

 

  • The leading Hemophilia A Companies includes Alnylam Pharmaceuticals, Sanofi Genzyme, Novo Nordisk, Catalyst Biosciences, Dimension Therapeutics, Chia Tai Tianqing Pharmaceutical Group, Staidson Beijing BioPharmaceuticals, Kymab, Poseida Therapeutics, Intellia Therapeutics, Regeneron Pharmaceuticals, Hoffmann-La Roche, Spark Therapeutics, BioMarin, Centessa Pharmaceuticals, Expression Therapeutics, Chugai Pharmaceutical, Pfizer, and others.

 

  • Promising Hemophilia A Pipeline Therapies includes Emicizumab, Biostate, ADVATE, ADYNOVI, PEGylated Recombinant Factor VIII, 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, NX-01 and others.

 

  • The Hemophilia A Companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The Hemophilia A pipeline therapies under development are focused on novel approaches to treat/improve Hemophilia A.

 

To explore more information on the latest breakthroughs in the Hemophilia A Pipeline treatment landscape of the report, click here @ Hemophilia A Pipeline Outlook

 

Hemophilia A Overview

Hemophilia A, also called factor VIII (8) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII (FVIII), a clotting protein. Hemophilia is inherited in an X-linked recessive manner. Hemophilia can vary in its severity, depending upon the particular type of mutation (genetic defect). The degree of symptoms depends upon the levels of the affected clotting factor. Symptoms of Hemophilia A include excessive bleeding from any site in the body along with long-term damage to joints from repeated bleeding episodes is characteristic. Diagnosis for suspected hemophilia include tests like Complete blood cell count, Screening coagulation studies, FVIII assay and FVIII inhibitor assay. Treatment goal for hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. Medication to treat hemophilia A is concentrated FVIII product. Several new treatments for hemophilia A are under development, from gene therapy to new non-factor replacement therapies.

 

Latest Developmental Activities in the Hemophilia A Treatment Landscape

 

  • The U.S. FDA has granted Fast Track Designation (FTD) for Efanesoctocog alfa, developed by Sanofi for Hemophilia A treatment.
  • In October 2021, Poseida Therapeutics and Takeda started working together to develop non-viral gene therapies for Hemophilia and other genetic diseases. Through a collaboration, Takeda Pharmaceutical will apply Poseida Therapeutics’ technologies to develop up to eight gene therapies including a Hemophilia A candidate under development by Poseida.
  • In January 2022, 2seventy bio entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for Hemophilia A. Under the terms of this agreement, Novo Nordisk will have the option to license 2seventy bio’s proprietary mRNA-based megaTAL™ technology for the development of a new treatment approach for Hemophilia A patients with the goal of offering a sustained therapeutic effect.
  • In March 2021, the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Valoctocogene roxaparvovec being developed by BioMarin. The company is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.
  • In November 2021, US FDA granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for Hemophilia A and B patients, with or without clotting factor inhibitors.
  • Giroctocogene fitelparvovec is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for Hemophilia A between Sangamo and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug (IND) application to Pfizer. The US FDA granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product Designation from the European Medicines Agency.

 

For further information, refer to the detailed Hemophilia A Unmet Needs, Hemophilia A Market Drivers, and Hemophilia A Market Barriers, click here for Hemophilia A Ongoing Clinical Trial Analysis

 

Hemophilia A Emerging Drugs Profile

 

Fitusiran: Alnylam Pharmaceuticals/Sanofi Genzyme

Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT). Alnylam Pharmaceuticals is developing the drug for the treatment of hemophilia and rare bleeding disorders (RBDs) in collaboration with Sanofi Genzyme. Fitusiran utilizes Alnylam Pharmaceuticals’ Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform. Fitusiran has been granted Orphan Drug Designation by FDA and Orphan Medicinal Product Designation by EMA for the treatment of hemophilia A and B.

 

Concizumab: Novo Nordisk

Concizumab is a high-affinity, humanized, anti–tissue factor (TF) pathway inhibitor (TFPI) based monoclonal antibody. The therapy is in clinical evaluation as a subcutaneously administered treatment for hemophilia A and B. 

 

Hemophilia A Pipeline Therapeutics Assessment

There are approx. 35+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Novo Nordisk.

 

Request a sample and discover the recent advances in Hemophilia A Ongoing Clinical Trial Analysis and Medications, click here @ Hemophilia A Treatment Landscape

 

Scope of the Hemophilia A Pipeline Insight Report

 

  • Coverage- Global

 

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

 

  • Hemophilia A Pipeline Therapies includes Emicizumab, Biostate, ADVATE, ADYNOVI, PEGylated Recombinant Factor VIII, 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, NX-01 and others.

 

  • Hemophilia A Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration

 

Dive deep into rich insights for drugs for Hemophilia A Market Drivers and Hemophilia A Market Barriers, click here @ Hemophilia A Unmet Needs and Analyst Views

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Hemophilia A: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Hemophilia A – DelveInsight’s Analytical Perspective
  7. In-depth Commercial Assessment
  8. Hemophilia A Collaboration Deals
  9. Late Stage Products (Phase III)
  10. Fitusiran: Alnylam Pharmaceuticals/Sanofi Genzyme
  11. Drug profiles in the detailed report…..
  12. Mid Stage Products (Phase II)
  13. Drug name: Company name
  14. Drug profiles in the detailed report…..
  15. Early Stage Products (Phase I/II)
  16. DTX 201: Dimension Therapeutics
  17. Drug profiles in the detailed report…..
  18. Preclinical and Discovery Stage Products
  19. KY1049: Kymab
  20. Drug profiles in the detailed report…..
  21. Inactive Products
  22. Hemophilia A Key Companies
  23. Hemophilia A Key Products
  24. Hemophilia A- Unmet Needs
  25. Hemophilia A- Market Drivers and Barriers
  26. Hemophilia A- Future Perspectives and Conclusion
  27. Hemophilia A Analyst Views
  28. Hemophilia A Key Companies
  29. Appendix

 

Got Queries? Find out the related information on Hemophilia A Mergers and acquisitions, Hemophilia A Licensing Activities @ Hemophilia A Emerging Drugs, and Recent Trends

 

 

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