Rewriting the Future OF LIVING WITH HEMOPHILIA B THROUGH GENE THERAPY—
BECOMING HIS BEST
Presented by
Growing up, Michael was always on the move. Whether it was basketball, golf, or swimming, he was the epitome of an active kid. But unlike other kids his age, he had to navigate life with a unique challenge.
Michael was diagnosed with hemophilia B at just 4 years old. Hemophilia B can cause prolonged bleeding and increases the risk for spontaneous bleeding and bleeding into joints due to the body not producing enough clotting factor IX. Every scrape or fall could result in a bleeding episode requiring treatment. For Michael, this usually meant a visit to the hospital for intravenous (into a person’s veins) infusions of factor IX replacement therapy, which are necessary to prevent or stop bleeding.
Tired of the constant visits to the hospital, Michael took a significant step by learning to manage his condition by self-infusing factor IX intravenously on a regular schedule in middle school. Self-infusing reduced the number of hospital visits, but breakthrough bleeding from simple accidents—like jamming his finger playing basketball—were still a constant worry.
“Any injury you sustain can be exacerbated. The fear of an injury or spontaneous breakthrough bleeding was always in the back of my mind,” he recalls.
Then as an adult, a worst-case scenario happened. Michael suffered a serious injury, tearing one of his pectoral muscles while lifting weights during a workout. “I had to undergo surgery, which was terrifying because hemophilia B puts me at a higher risk of bleeding during the operation and recovery,” he said. Michael had to rely heavily on his family during this time as he had to increase his self-infusions. While Michael recovered without any serious complications, the constant fear of bleeding and the need to adhere to a strict infusion schedule took a toll on him physically and mentally.
Michael had already started learning from his care team about HEMGENIX® (etranacogene dezaparvovec-drlb)—a one-time infusion that can elevate factor IX levels for years, offering greater bleed protection and significantly reducing or eliminating the need for regular factor IX infusions—and while he was already considering it, the experience solidified the decision.
“I’ve had needles stuck in me multiple times a month since I was 4-years old and the potential to not need regular infusions anymore was absolutely life changing,” Michael said.
Following pre-screening tests and securing approval from his insurance company, in March 2024 Michael received HEMGENIX. “For me, the process was simple. The infusion took about an hour and then I had to stay for about three hours for monitoring for infusion-related reactions. My grandmother and I then simply drove back home.”
Following his infusion, Michael was monitored for side effects such as headaches, flu-like symptoms, fatigue, nausea, or feeling unwell, and had several follow-up appointments to monitor his liver and blood enzyme levels, as well as his factor IX levels. Michael’s body began to produce an increased amount of
factor IX, which it couldn’t do before. The best part he says is that due to the increased factor IX, he hasn’t experienced any breakthrough bleeds since receiving HEMGENIX and he no longer needs regular infusions.
“I’ve noticed that I feel more confident and less stressed, not worrying when I exercise and play basketball if I need to self-infuse before or after because I know my factor IX levels are steady,” he said.
HEMGENIX has given Michael a newfound sense of freedom. He looks forward to earning his MBA and spending more time doing the things he loves, with less worry. “Continuing to talk to my healthcare team about the challenges I was having led to a new treatment for me. I encourage others to do the same to find what’s right for them.”
Michael working out weeks after his HEMGENIX infusion. Source: CSL Behring
For more information on gene therapy for hemophilia B, visit https://www.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, or
• Have current or historical life-threatening bleeding, or
• Have 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 of CSL Behring LLC.
©2024 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA USA-HGX-0915-DEC24
Presented by
Rewriting the Future OF LIVING WITH HEMOPHILIA B THROUGH GENE THERAPY—
BECOMING HIS BEST
Growing up, Michael was always on the move. Whether it was basketball, golf, or swimming, he was the epitome of an active kid. But unlike other kids his age, he had to navigate life with a unique challenge.
Michael was diagnosed with hemophilia B at just 4 years old. Hemophilia B can cause prolonged bleeding and increases the risk for spontaneous bleeding and bleeding into joints due to the body not producing enough clotting factor IX. Every scrape or fall could result in a bleeding episode requiring treatment. For Michael, this usually meant a visit to the hospital for intravenous (into a person’s veins) infusions of factor IX replacement therapy, which are necessary to prevent or stop bleeding.
Tired of the constant visits to the hospital, Michael took a significant step by learning to manage his condition by self-infusing factor IX intravenously on a regular schedule in middle school. Self-infusing reduced the number of hospital visits, but breakthrough bleeding from simple accidents—like jamming his finger playing basketball—were still a constant worry.
Then as an adult, a worst-case scenario happened. Michael suffered a serious injury, tearing one of his pectoral muscles while lifting weights during a workout. “I had to undergo surgery, which was terrifying because hemophilia B puts me at a higher risk of bleeding during the operation and recovery,” he said. Michael had to rely heavily on his family during this time as he had to increase his self-infusions. While Michael recovered without any serious complications, the constant fear of bleeding and the need to adhere to a strict infusion schedule took a toll on him physically and mentally.
Michael had already started learning from his care team about HEMGENIX® (etranacogene dezaparvovec-drlb)—a one-time infusion that can elevate factor IX levels for years, offering greater bleed protection and significantly reducing or eliminating the need for regular factor IX infusions—and while he was already considering it, the experience solidified the decision.
“I’ve had needles stuck in me multiple times a month since I was 4-years old and the potential to not need regular infusions anymore was absolutely life changing,” Michael said.
Following pre-screening tests and securing approval from his insurance company, in March 2024 Michael received HEMGENIX. “For me, the process was simple. The infusion took about an hour and then I had to stay for about three hours for monitoring for infusion-related reactions. My grandmother and I then simply drove back home.”
Following his infusion, Michael was monitored for side effects such as headaches, flu-like symptoms, fatigue, nausea, or feeling unwell, and had several follow-up appointments to monitor his liver and blood enzyme levels, as well as his factor IX levels. Michael’s body began to produce an increased amount of factor IX, which it couldn’t do before. The best part he says is that due to the increased factor IX, he hasn’t experienced any breakthrough bleeds since receiving HEMGENIX and he no longer needs regular infusions.
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, or
• Have current or historical life-threatening bleeding, or
• Have 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 of CSL Behring LLC.
©2024 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA USA-HGX-0915-DEC24