Hemophilia B
A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)
Drugs by status
Approved for this indication (12)
- coagulation factor ix (Recombinant)
- concizumab
- human factor ix complex (prothrombin complex concentrate)
- Marstacimab
- factor ix albumin fusion protein
- coagulation factor viia (recombinant)
- coagulation factor ix (Recombinant), Fc-fusion protein
- fidanacogene elaparvovec
- coagulation factor xiii a-subunit (recombinant)
- human coagulation factor ix
- albutrepenonacog alfa
- factor xiii concentrate (human)
Phase 4 trials (2)
Phase 3 trials (14)
Phase 2 trials (12)
Phase 1 trials (10)
Organizations
Organization Involved with Phase 4 Indications (8)
Organization Involved with Phase 3 Indications (18)
- CSL Behring
- Swedish Orphan Biovitrum
- Catalyst Biosciences
- Aptevo Therapeutics
- Bio Products Laboratory
- Cangene
- Genzyme
- Sanofi
- Biogen
- Bayer
- Bioverativ Therapeutics Inc.
- Laboratoire français de Fractionnement et de Biotechnologies
- University of Michigan
- University of Colorado, Denver
- University of Montpellier
- UniQure N.V.
- LFB Biotechnologies, SAS
- Freeline Therapeutics
Organization Involved with Phase 2 Indications (19)
- Avigen
- Grifols
- Alnylam Pharmaceuticals
- Chiesi
- Dimension Therapeutics
- PTC Therapeutics
- Spark Therapeutics
- National Center for Research Resources (NCRR)
- University of Pennsylvania
- University of Pittsburgh
- University of Washington
- Stanford University
- University of Sydney
- University of Campinas
- Opko
- University of Texas at Houston
- University of North Carolina at Chapel Hill
- Christian Medical College, Vellore, India
- Centessa
Organization Involved with Phase 1 Indications (10)
Organization Involved with Other Experimental Indications (3)