The FDA has accepted CSL Behring’s priority review application for a hemophilia B gene therapy, potentially fast-tracking the drug after a bumpy ride that included a clinical hold—and later, a lift.
If approved, etranacogene dezaparvovec—an AAV-5 gene therapy candidate given as a one-time treatment—would be the first gene therapy option for patients with hemophilia B, a life-threatening degenerative disease.
CSL Behring, the global biotherapeutics business of Australia-based CSL, acquired the gene therapy from biotech uniQure in June 2020. The therapy is designed to significantly reduce blood-clotting by addressing the underlying root of hemophilia B: a faulty gene that leads to a clotting deficiency.
