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