Advancements in Hemophilia A and B treatments are progressing with multiple companies developing novel therapies. These treatments range from recombinant factors to gene therapies, offering new hope for patients with these bleeding disorders.
Clinical Trial Updates
TiumBio Co., Ltd. recently announced the submission of a Clinical Trial Application (CTA) to the Italian Medicines Agency (AIFA) and the Spanish Agency of Medicines and Medical Products (AEMPS) for TU7710. TU7710 is a novel recombinant activated factor VII (rFVIIa) intended for hemophilia patients who have developed inhibitors. The Phase 1b trial aims to evaluate its safety and efficacy in this challenging patient population.
Centessa Pharmaceuticals has initiated dosing in a Phase 2b clinical trial (PRESent-2, NCT05789524) to assess SerpinPC, an experimental treatment for hemophilia B. This open-label trial is part of the company's registration program, designed to gather evidence for potential approval.
Recent Approvals
In February 2023, the U.S. Food and Drug Administration (FDA) approved ALTUVIIIO (efanesoctocog alfa), a factor VIII replacement therapy developed by Sanofi, for both adults and children with hemophilia A. ALTUVIIIO is indicated for routine prophylaxis to prevent bleeding episodes, on-demand treatment of bleeding episodes, and perioperative management.
BioMarin announced in June 2023 that the FDA had approved Roctavian (valoctocogene roxaparvovec), a single-dose gene therapy for severe hemophilia A. This approval marks a significant milestone, offering a potentially curative treatment option for patients with this condition.
Pipeline Overview
Numerous companies are actively involved in developing therapies for hemophilia A and B. Key players include:
- Novo Nordisk
- Regeneron Pharmaceuticals
- Shanghai Belief-Delivery BioMed
- Biocad
- Pfizer
- CSL Behring
- Baxalta (now part of Shire)
- ApcinteX Ltd
- Staidson (Beijing) Biopharmaceuticals
- Genzyme
- Suzhou Alphamab
- Sanofi
- Jiangsu Gensciences lnc.
- Spark Therapeutics, Inc.
- Bayer
- BioMarin Pharmaceutical
- Ultragenix Pharmaceutical
- Sigilon Therapeutics
- Expression Therapeutics, LLC
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd
- Generation Bio
- 2seventy bio
- Sinocelltech Ltd
- Hoffmann-La Roche
- Chugai Pharmaceutical
- ASC Therapeutics
- Poseida Therapeutics
- Apitope Technology
These companies are developing a range of therapies, including:
- Nonacog beta pegol
- REGV131
- BBM-H901
- ANB-002
- PF-06838435
- AAV5-hFIXco-Padua
- AskBio009
- Marstacimab
- SerpinPC
- Concizumab
- STSP-0601
- Fitusiran
- KN057
- SS109
- SPK-8011
- BAY2599023 (DTX201)
- Efanesoctocog alfa
- Valoctocogene roxaparvovec
These therapies are in various stages of clinical development, from Phase 1 to Phase 3, and utilize different mechanisms of action, including factor replacement, gene therapy, and bispecific antibodies.
Hemophilia Overview
Hemophilia is a rare genetic bleeding disorder characterized by a deficiency in clotting factors, leading to prolonged bleeding. Hemophilia A is caused by a deficiency in factor VIII, while hemophilia B is caused by a deficiency in factor IX. The severity of hemophilia varies depending on the level of clotting factor deficiency.
