Fitusiran
Published , updated
Unassigned
New Medicines
Moderate to severe haemophilia A and B in adults
Information
New molecular entity
Sanofi
Sanofi
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes
Category
RNA interference (RNAi) therapeutic targeting antithrombin (AT)
Haemophilia A is defined by loss-of-function mutations in Factor VIII, and there are greater than 40,000 registered patients in the U.S. and E.U. Haemophilia B, defined by loss-of-function mutations in Factor IX, affects greater than 9,500 registered patients in the U.S. and E.U. [1]
Moderate to severe haemophilia A and B in adults
Subcutaneous
Haemophilia A and B in children
Information
Licence extension / variation
Sanofi
Sanofi
Development and Regulatory status
None
Phase III Clinical Trials
None
Yes
Yes
Category
RNA interference (RNAi) therapeutic targeting antithrombin (AT)
Haemophilia A is defined by loss-of-function mutations in Factor VIII, and there are greater than 40,000 registered patients in the U.S. and E.U. Haemophilia B, defined by loss-of-function mutations in Factor IX, affects greater than 9,500 registered patients in the U.S. and E.U. [1]
Haemophilia A and B in children
Subcutaneous