CSL Behring announced that the first patient has received Hemgenix® (etranacogene dezaparvoved-drlb), the only gene therapy currently approved by the US Food and Drug Administration (FDA) to treat adults with hemophilia B who are on IX prophylaxis or who have chronic, serious bleeds.
The announcement, via news release, noted that etranacogene dezaparvoved-drlb is a paradigm-shifting therapy for patients with hemophilia from strictly scheduled infusions to a single, one-time injection.
“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,” wrote Matthew Ryan, MD, adult hematologist and medical director at Hemophilia Outreach Center, Green Bay, Wisconsin.
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Hemophilia B is a rare, life-threatening condition caused by a mutation on the F9 gene, leading to insufficient levels of factor IX, which helps blood clots form.
Read more about hemophilia therapies
Etranacogene dezaparvoved-drlb uses AAV5, a noninfectious viral vector, to deliver a gene variant of factor IX to target liver cells, enabling them to produce highly active factor IX proteins that can generate stable levels of factor IX for years.
Clinical trials have shown that etranacogene dezaparvoved-drlb can increase and maintain factor IX levels while also reducing annual bleed rates and the need for prophylactic treatments in almost all (94%) of patients treated. In addition to the benefits for patients, healthcare systems could see significant cost savings given that for patients with hemophilia B, their costs can be up to 25 times higher than for individuals without a bleeding disorder.
As of May 2023, health insurance companies covering approximately 60% of the US population have established policies for covering etranacogene dezaparvoved-drlb treatment.
Reference
CSL Behring announces the first patient has received FDA-approved HEMGENIX® (etranacogene dezaparvovec-drlb) for hemophilia B. News release. CSL Behring; June 20, 2023.
