CSL Behring has announced that the first patient has been dosed with HEMGENIX®, the only hemophilia B gene therapy product currently approved in the U.S.

Approved by the U.S. Food and Drug Administration in November 2022, HEMGENIX is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. Clinical trial development of the therapy was led by uniQure, while global rights to commercialize the product are held by CSL Behring.

"As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone of the first commercial patient having received HEMGENIX and look forward to continuing to deliver this paradigm-shifting treatment to the hemophilia B community," said Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring. "To the advocacy organizations, medical experts, healthcare professionals, payers, channel partners, and the entire hemophilia community who have collaborated with us to establish access for this innovative therapy, we thank you and look forward to many more patients benefiting from this therapy."

"Discussions between CSL Behring and the payer community on the value proposition offered for HEMGENIX have been positive, which is reflected in the policies that have been written," added Lojewski. "As of May 2023, payers covering roughly 60 percent of the U.S. population have established clear medical policies covering HEMGENIX. For those who have not yet established policies, we anticipate that coverage decisions for HEMGENIX will be reviewed on a case-by-case basis."

Source: CSL Behring press release dated June 20, 2023