Fitusiran
Unassigned
New Medicines
Moderate-to-severe haemophilia A and B
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
Subcutaneous