QURE - CSL uniQure's gene therapy for hemophilia B gets FDA priority review

The U.S. Food and Drug Administration (FDA) granted priority review to uniQure (NASDAQ:QURE) and CSL's (OTCPK:CSLLY) gene therapy etranacogene dezaparvovec (EtranaDez) to treat hemophilia B. The FDA accepted to review a biologics license application (BLA) submitted by CSL Behring, a unit of Australia's CSL. Under priority review the FDA expects to make a decision within six months, compared to 10 months for standard review. 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 would be the first ever gene therapy treatment option for hemophilia B. The company noted that the BLA was backed by data from a trial called HOPE-B. The gene therapy is also under review in the EU.

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CSL, uniQure's gene therapy for hemophilia B gets FDA priority review