dm+d

Unassigned

New Medicines

Moderate to severe haemophilia A and B in adults and children aged 12 years and older

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 and children aged 12 years and older
Subcutaneous

Haemophilia A and B in children aged less than 12 years

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 aged less than 12 years
Subcutaneous