valoctocogene roxaparvovec-rvox
Pfizer’s sickle cell disease therapy Oxbryta recommended by NICE for NHS use
About 4,000 people in England with the genetic blood disorder are expected to benefit from the decision
BioMarin shares long-term results for haemophilia A gene therapy Roctavian
Approximately one in 10,000 people are affected by the genetic blood clotting disorder
BioMarin announces first patient dosed with haemophilia A gene therapy in Europe
The therapy was granted conditional marketing authorisation by the EC in August 2022
FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A
Approximately 2,500 patients are expected to be eligible to receive the one-time therapy
EC approves BioMarin’s gene therapy Roctavian for severe haemophilia A
Approximately 20,000 adults are affected by haemophilia A across more than 70 countries in Europe, the Middle East and Africa