First Hemophilia B Patient Receives FDA-Approved HEMGENIX® Treatment by CSL Behring

Tuesday, June 20, 2023

CSL Behring, a renowned global biotechnology leader, has announced a significant milestone in the United States. The first patient in the country has received HEMGENIX® (etranacogene dezaparvovec-drlb), an FDA-approved gene therapy for hemophilia B. This groundbreaking treatment is specifically designed for adults with hemophilia B who currently rely on factor IX prophylaxis therapy, experience life-threatening bleeding, or have repeated serious spontaneous bleeding episodes.

Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring, expressed his enthusiasm for achieving this milestone and reiterated the company's dedication to delivering this transformative treatment to the hemophilia B community. He extended gratitude to advocacy organizations, medical experts, healthcare professionals, payers, channel partners, and the entire hemophilia community for their collaborative efforts in making this innovative therapy accessible.

Despite advancements in prophylactic treatments for hemophilia B, which have proven effective, there is still the need for strict infusion schedules, and the risk of breakthrough bleeds into joints persists. HEMGENIX has the potential to revolutionize the treatment approach by directly addressing the underlying genetic cause of the condition through a single, one-time infusion.

Clinical trials have demonstrated that HEMGENIX can elevate and sustain factor IX levels for extended periods, significantly reduce the annual bleed rate compared to standard care, and eliminate or minimize the need for prophylactic treatment in 94% of patients. In addition to the long-term health benefits of improved bleed protection and freedom from infusion schedules, HEMGENIX also has the potential to generate significant cost savings for the healthcare system. The costs associated with hemophilia B can be up to 25 times higher compared to individuals without bleeding disorders, resulting in a total lifetime cost exceeding $20 million per person.

CSL Behring has engaged in positive discussions with payers to establish the value proposition of HEMGENIX, leading to the development of clear medical policies by payers covering approximately 60% of the U.S. population. For payers who have yet to establish policies, coverage decisions for HEMGENIX will be considered on a case-by-case basis.

Dr. Matthew Ryan, Adult Hematologist and Medical Director at Hemophilia Outreach Center in Green Bay, WI, emphasized the significance of HEMGENIX as a major advancement for the hemophilia B community. He highlighted the transformative impact of this treatment, offering patients the opportunity to live without frequent bleeds or the burden of regular infusions. Moreover, he stressed the importance of ongoing discussions within the community regarding treatment options and goals, considering the evolving needs of individuals with this lifelong condition.

Patients who choose to pursue treatment with HEMGENIX, in consultation with their healthcare providers, can participate in the HEMGENIX ConnectSM program. This program provides patients with a dedicated support team, including a Patient Resource Navigator and CSL Case Manager, who can address their inquiries related to gene therapy, the treatment process, and insurance coverage.

As part of their commitment to the hemophilia B community, CSL Behring continues to provide training to medical centers that may administer HEMGENIX. They also encourage the collection of long-term data through the ATHN registry, which will provide valuable insights to all stakeholders and generate additional evidence regarding the long-term safety, efficacy, and durability of gene therapy.

Apart from FDA approval in the United States, HEMGENIX has also received approval from the European Commission (EC) for the European Union and European Economic Area. Furthermore, it has obtained conditional marketing authorization from the United Kingdom's Medicines and Healthcare product Regulatory Agency. The development of HEMGENIX involved a multi-year collaboration between CSL Behring and uniQure, with CSL Behring acquiring global rights for commercialization.

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