CSL's gene therapy for haemophilia B has been recommended for approval by the EMA's human medicine committee, setting up a decision by the European Commission early next year.
uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy fo
Dutch biotech uniQure and partner CSL Behring are planning to file for approval of their gene therapy for haemophilia B, armed with new trial data that they say answers earlier concerns abo
Sanofi's much anticipated drug for both haemophilia A and B – fitusiran – could be filed in 2022 despite a problem with blood clots in some patients in late-stage testing.
Dosing can start once again in uniQure's phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by
