Valoctocogene roxaparvovec

Unassigned

New Medicines

Severe haemophilia A in males

Information

Advanced therapy medicinal product (ATMP)
BioMarin
BioMarin

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

A gene therapy, designated as BMN 270, to stimulate the production of factor VIII, a blood coagulation factor, for the treatment of haemophilia A.
It affects 1:4,000 to 1:5,000 live male births worldwide [1].
Severe haemophilia A in males
Intravenous infusion