Gene therapy offers people with rare bleeding disorder a new level of freedom

(BPT) – -Hemophilia B by the NumbersHemophilia B affects ~6,000 people in the U.S., and up to two-thirds have a moderate to severe version of the condition.Some people with hemophilia B may require up to 156 intravenous infusions per year.43% of people living with hemophilia B experience depression, anxiety or other psychological disorders.95% report the condition negatively impacts their employment.Overall total annual healthcare costs for people with hemophilia can be 25x higher compared to individuals who do not have a bleeding disorder.Imagine you have a rare bleeding disorder and have spent much of your life knowing you are at greater risk of uncontrollable bleeding. You self-infuse treatment on a strict, regular schedule to make sure your blood will clot. This restrictive infusion schedule can impact work, education, lifestyle, and even where you vacation.Now, imagine a treatment option that can offer consistent bleed protection for years after a one-time infusion, lowering the risk of bleeds and reducing the burden you’ve experienced as a person with a rare bleeding disorder. This is now a reality for some people with hemophilia B after the U.S. Food and Drug Administration (FDA) approved HEMGENIX® (etranacogene dezaparvovec-drlb), the first and only gene therapy for 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.The future of hemophilia B treatment is nowHemophilia B is a rare bleeding disorder that affects around 6,000 people in the U.S. For people with the condition, their blood does not clot in the typical way because they lack sufficient factor IX, a protein primarily produced by the liver that helps blood clots form.The more severe the condition, the less able a person is to form blood clots, making them more vulnerable to bleeding. If not adequately controlled, people with the condition are more vulnerable to trauma and spontaneous bleeding into their joints. Sometimes these bleeds can be life-threatening and can lead to permanent physical damage. The most common treatment for people with moderate to severe hemophilia B is lifelong prophylactic infusions to temporarily replace or supplement low levels of blood-clotting factor.But with HEMGENIX, people with the condition can produce their own elevated and sustained levels of factor IX for years following a one-time single intravenous infusion. Now, people with hemophilia B are beginning to receive this innovative treatment at hemophilia treatment centers across the U.S.How—and why—HEMGENIX breaks new ground for people with hemophilia BHEMGENIX uses a gene therapy approach called gene transfer. This approach aims to introduce a working gene into the body to produce functional factor IX and support natural blood-clotting ability.The working gene is loaded into an inactive viral shell, known as a vector. HEMGENIX uses an adeno-associated viral vector serotype 5, an AAV5 vector. Administered as a one-time IV infusion, HEMGENIX heads toward the liver with the working gene. Once it arrives in the liver, the working gene’s instructions are unloaded and the AAV vector shell is broken down and eliminated. The genetic instructions remain, however, so that the liver can produce elevated and protective factor IX on its own. Following administration of HEMGENIX, overall health, liver health, and factor IX activity levels should be tracked weekly for the first three months so the doctor can monitor the response to HEMGENIX.Results from clinical trials of HEMGENIX showed people treated with the one-time infusion produced a mean factor IX activity of 37% and had a significant reduction in annual bleed rates. Encouragingly, 94% of people (51 out of 54) discontinued routine factor IX prophylaxis. The most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell.“I am proud to have been involved in a clinical trial program that had such good results and offers so much hope to the hemophilia B community,” said Dr. Steven Pipe, professor at the University of Michigan, Ann Arbor, and medical director of the Pediatric Hemophilia and Coagulation Disorders Program. “As I follow those who have gone through this treatment and see their lives blossom, I continue to be excited. I have a clinical trial participant who says he celebrates two birthdays—the day of his official birth and the date he received treatment.”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.Talk with your doctorPeople living with hemophilia B who are interested in HEMGENIX should talk to their doctor about the potential benefits and eligibility screening.Those who decide with their doctor to move forward with treatment can enroll in HEMGENIX ConnectSM where they will be assigned a dedicated support team, including a Patient Resource Navigator and CSL Case Manager, who can:Provide education on gene therapy and how it worksProvide support and guidance on the HEMGENIX treatment journeyAssist with insurance questions and review, explaining benefits and determining eligibility for financial assistance programsGet support Having a rare disease can feel isolating and trying a new treatment can cause a mix of emotions, from excitement to anxiety. The B SupportTM app was designed specifically to help people with hemophilia B as they explore whether gene therapy is right for them.The app allows people with hemophilia B to:Record bleeds, factor IX activity and how they are feeling to better track their current treatment and progress over time. Having this information at their fingertips, either on their phone or tablets, allows people with hemophilia B to have robust conversations with their healthcare professional about whether they are meeting their treatment goals, and if needed, other therapeutic options.Get valuable resources, reminders and support to enhance their experience managing hemophilia B.Access education on gene therapy, including information on eligibility.The app is available in the Apple and Google Play stores in the U.S.With advanced research and new treatments, people with hemophilia B may have more freedom from their treatment schedule. To learn more, visit HEMGENIX.com.Important Safety Information?What is HEMGENIX?HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:Currently use Factor IX prophylaxis therapy, orHave current or historical life-threatening bleeding, orHave repeated, serious spontaneous bleeding episodes.HEMGENIX is administered as a single intravenous infusion and can be administered only once.What medical testing can I expect to be given before and after administration of HEMGENIX?To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.What were the most common side effects of HEMGENIX in clinical trials?In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.What should I watch for during infusion with HEMGENIX?Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.What should I avoid after receiving HEMGENIX?Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.Please see full prescribing information for HEMGENIX. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.?HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.HEMGENIX® is a registered trademark and B SupportTM is a trademark of CSL Behring LLC.HEMGENIX ConnectSM is a service mark of CSL Behring LLC.©2023 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USAUSA-HGX-0456-JUL23