Dear Hemophilia B Community,
We are writing to share an important update on the availability of HEMGENIX® (etranacogene dezaparvovec-drlb).
CSL is currently experiencing a temporary global stockout of HEMGENIX® that will result in delays in treatment for some individuals in countries with established commercial access.
We want to be very clear that this situation is not related to the safety or effectiveness of HEMGENIX®. Rather, it reflects the complexity of manufacturing gene therapies, and our commitment to adhering to the highest regulatory and quality standards for the people we serve. We are working with regulatory authorities on strategies to ensure stable ongoing supply for HEMGENIX® while preserving our high-quality standards.
We know that this update may raise questions and that’s completely understandable. What we want you to know is that our highest priority, as always, is ensuring that eligible individuals have the information they need to determine if HEMGENIX® is right for them, and that when they do, HEMGENIX® is available in accordance with the highest standards. We remain fully committed to delivering this innovative, one-time gene therapy to the hemophilia B community and continue to have strong confidence in HEMGENIX®.
We understand the careful thought and planning that go into every individual decision about gene therapy treatment. We deeply value our long‑standing partnership of trust with the hemophilia B community and will continue to keep you updated to ensure people with hemophilia B and their healthcare providers are informed and supported as they plan their hemophilia care needs.
For further information, please visit our contact page on CSL.com here: https://www.csl.com/contact.
Sincerely,
Dr. Deborah Long,
SVP, Medical Affairs
Important Safety Information (ISI)
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.