Real-world results with hemophilia B gene therapy
The following content is provided by CSL Behring LLC and does not reflect the views of BioNews, Inc.
A game changer for patients with hemophilia B
Andrea was in her current role as a nurse practitioner at a Wisconsin hemophilia treatment center when she first heard about HEMGENIX® (etranacogene dezaparvovec-drlb), and she was impressed. FDA approved in 2022 and studied since 2018, it caught her attention as a promising single-infusion treatment for adult male hemophilia B patients that enables the liver to make its own factor IX.
“There are a lot of thoughts that go through your mind that this is amazing. It’s novel.”
Andrea was excited about the potential impact HEMGENIX could have on her patients. Three-year data published in December 2023 show patients maintained elevated and sustained factor IX levels for years, and that the majority were able to discontinue routine prophylaxis (prophy). Furthermore, the safety of HEMGENIX has also been studied since 2018, giving her even more reason to be hopeful for positive outcomes.
“It was really just digging into all the clinical data to make sure that before we offer this as a treatment plan, that it’s something we feel is going to be safe and [effective] for our patients.”
Also driving the decision was the need—and demand—for a treatment that could provide even better bleed protection than prophy. And because HEMGENIX is a single-infusion therapy, Andrea felt it’s ideal for those who have difficulty maintaining their prophy schedule.
“We were looking at those patients that are either still having bleeds while on prophylaxis or their lifestyle did not allow for prophylaxis, or they weren’t the most compliant with prophylaxis. And we said, you know, these are patients that we think would really benefit from HEMGENIX.”
“They’re coming to us to say, ‘Hey, tell me about [HEMGENIX].’”
Zeroing in on the right patients for this initial infusion proved easy, as they were young, had issues with prophy resulting from a fear of needles, and were highly motivated and well-informed about HEMGENIX and what it could possibly mean for their lives.
“[They] were very excited to know that there is this treatment option that could decrease the number of pokes that they were hoping to not have to do anymore.”
Since this was the center’s first HEMGENIX administration, prepping for it took about 4 months. Blood work and liver scans were required to ensure the patients responded favorably. To help keep the process moving forward, insurance coverage was verified at the same time. As for the actual infusion day, it went exceptionally well, according to Andrea, and was met with lots of excitement on the part of the patients, their families, and the staff.
“You have probably never seen more excited faces on the day of infusion. I think we could not have asked for a better day. [The] infusions went fantastically. And when they finished and their monitoring was done, … as you’ve probably seen in the pictures, there were lots of hands in the air and cheers that took place.”
For Andrea’s patients, HEMGENIX resulted in elevated factor IX levels and no more prophy
A successful infusion day was followed by equally successful results. Early responders to HEMGENIX, Andrea’s patients experienced elevated factor IX levels after the first week and were able to stop routine prophy after week 2 with zero bleeds, even following minor injuries. One year later, they’re still doing very well.
“They have not had to do any factor IX infusions, since they’ve not had any bleeds. This has truly made an impact on their lives. When they come in for their lab draws, you see the smiles on their faces still. They will tell you hands down, it was the best decision that they’ve made.”
HEMGENIX Connect℠ helped smooth the way
Andrea’s facility and the patients were able to rely on HEMGENIX Connect to handle what can often be a difficult process. Their dedicated team assisted with benefit investigations, prior authorizations, letters of medical necessity, and travel arrangements to and from the treatment facility.
“I think, in general, the HEMGENIX process is very well designed by CSL Behring. We had a lot of help along the way from the team and were able to reach out at any time. The HEMGENIX Connect program was very beneficial from the patient perspective and the fact that they live a distance from our center.”
Ready to try HEMGENIX?
For those seriously considering HEMGENIX but also questioning whether it’s right for them, Andrea recommends that they think about what their goals are for this treatment. Having an open and honest conversation with their doctor may also help. Additionally, knowing that HEMGENIX is covered by most US health insurance companies and that most commercially insured patients have a $0 out-of-pocket cost might ease their decision.
“It’s really talking with every patient about … what their goals are for themselves and their bleeding disorder and just making sure that they know that there is this option out there that could help them meet those goals and ensuring that they’re well educated.”
What about doctors and other healthcare professionals (HCPs)?
For HCPs who have questions or are hesitant to offer gene therapy, Andrea encourages them to talk to colleagues who have experience administering HEMGENIX based on the promising results she’s seen so far. Patients can also express their interest and ask their doctors to start the prescreening process.
“Just that joy you see on a patient when you tell them that they’re making their own factor IX is well worth all the work you put into it.”
Let’s do this!
The first step to starting treatment with HEMGENIX is to talk to your doctor and ask them to begin the screening process to confirm your eligibility. If your doctor isn’t ready to administer HEMGENIX, you can use the locator tool to find other administration centers that are trained and prepared. For questions and support throughout the treatment process, including assistance with reimbursement and insurance, be sure to enroll in HEMGENIX Connect.
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.
HEMGENIX Connect℠ is a service mark of CSL Behring LLC.
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