QURE - CSL uniQure's gene therapy for hemophilia B under review in EU

The European Medicines Agency accepted a marketing authorization application by CSL's (OTCPK:CSLLY) unit CSL Behring's for gene therapy etranacogene dezaparvovec (EtranaDez) to treat hemophilia B. Etranacogene dezaparvovec is administered as a one-time treatment for patients with hemophilia B, a genetic bleeding disorder. The company said that if approved, etranacogene dezaparvovec will be the first-ever gene therapy treatment option that significantly reduces the rate of annual bleeds after a single infusion. The application is backed by data from a trial called HOPE-B. The multi-year clinical development was led by uniQure (NASDAQ:QURE) and sponsorship of the trials in the U.S. has transitioned to CSL Behring after its acquiring global rights to commercialize etranacogene dezaparvovec. CSL Behring said it is in the process of transitioning sponsorship of the trials in the EU.

For further details see:

CSL, uniQure's gene therapy for hemophilia B under review in EU