KING OF PRUSSIA, Pa., June 20, 2023 /PRNewswire/ -- Global biotechnology leader CSL Behring today announced that the first patient has received U.S. Food and Drug Administration (FDA) approved HEMGENIX^® (etranacogene dezaparvovec-drlb) for hemophilia B in the United States. HEMGENIX is the first and only gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes. 

"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."

While prophylactic hemophilia B treatments have significantly advanced over the past several decades and are effective, treatments are typically administered on a strict infusion schedule and breakthrough bleeds into joints are still possible. HEMGENIX has the potential to fundamentally transform the treatment paradigm through a single, one-time infusion in people living with hemophilia B by addressing the genetic cause of the condition.

In clinical trials, HEMGENIX demonstrated the ability to elevate and sustain factor IX levels for years, significantly reduce the rate of annual bleeds versus standard of care, and reduce or eliminate the need for prophylactic treatment in 94% of people who received the therapy. In addition to the potential long-term health benefits from greater bleed protection and liberation from infusion schedules, HEMGENIX could also generate significant cost savings for the healthcare system at-large. Healthcare costs can be 25 times higher for a person living with hemophilia B compared to individuals who do not have a bleeding disorder—amounting to a total adult lifetime cost of more than $20 million per person.

"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."

"HEMGENIX is an important addition to the current treatment paradigm and a long-awaited advancement for the hemophilia B community," said Matthew Ryan, MD, Adult Hematologist and Medical Director at Hemophilia Outreach Center, Green Bay, WI. "This exciting treatment offers patients a chance to live a life without frequent bleeds or the burden of regular infusions. The availability of this new one-time treatment also sparks additional conversations in the community when it comes to treatment options and goals, which is extremely important when managing a life-long condition, as an individual's needs can change for a variety of reasons."

People living with hemophilia B who have decided with their doctor to move forward with treatment with HEMGENIX, can enroll in the HEMGENIX Connect^SM program where they will be assigned a dedicated support team, including a Patient Resource Navigator and CSL Case Manager, who can assist with questions about gene therapy, the treatment journey and insurance coverage.

In further demonstration of the company's commitment to the hemophilia B community, CSL Behring is providing ongoing training to centers who may administer HEMGENIX and encouraging long term data collection through the ATHN registry. Registry data will be informative to all stakeholders and will generate additional evidence on the long-term safety, efficacy, and durability of gene therapy.

HEMGENIX was also approved by the European Commission (EC) for the European Union and European Economic Area and was granted conditional marketing authorization by the United Kingdom's Medicines and Healthcare product Regulatory Agency. The multi-year clinical development of HEMGENIX was led by uniQure (Nasdaq: QURE) and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment.

About Hemophilia B
Hemophilia B is a life-threatening rare disease caused by a mutation on the F9 gene, resulting in low levels of functional clotting factor IX. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor. 

About HEMGENIX^®
HEMGENIX^® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.

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Media Contact
Etanjalie Ayala       
Mobile: +1 610 297 1069
Email: etanjalie.ayala@cslbehring.com