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EMA starts Review of Gene Therapy for Haemophilia B

EMA 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 indicates the therapeutic effect of a single infusion lasts for years. 
  • Milestone underlines CSL Behring’s promise to develop and deliver a truly unique portfolio of patient-focused therapies.