
European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B
Marketing Authorization Application (MAA) for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to be the first gene therapy for patients living with hemophilia B. MAA filing is supported by the phase 3 HOPE-B study which demonstrated durable and sustained therapeutic effect after a single infusion.