Gene Therapy

CSL Behring has announced that the first patient in the United States has received HEMGENIX®, which is currently the only approved gene therapy product for hemophilia B. HEMGENIX was approved by the U.S. Food and Drug Administration in November 2022 and is intended for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have experienced life-threatening hemorrhages or frequent and severe spontaneous bleeding episodes. The development of this therapy was led by uniQure, while CSL Behring holds the global rights to commercialize the product.

Bob Lojewski, Senior Vice President and General Manager of North America at CSL Behring, expressed excitement about reaching this milestone and emphasized the company's commitment to providing this groundbreaking treatment to the hemophilia B community. He thanked advocacy organizations, medical experts, healthcare professionals, payers, channel partners, and the entire hemophilia community for their collaboration in ensuring access to this innovative therapy. CSL Behring looks forward to benefiting many more patients with HEMGENIX.

CSL Behring has had positive discussions with the payer community regarding the value of HEMGENIX, resulting in the formulation of supportive policies. As of May 2023, approximately 60% of the U.S. population is covered by payers with clear medical policies for HEMGENIX. For those payers who have yet to establish policies, coverage decisions for HEMGENIX will be evaluated on an individual basis.

Source: CSL Behring press release dated June 20, 2023