SAFETY PROFILE FOR HEMGENIX
HEMGENIX was found safe and effective, with no serious treatment-related side effects reported.*
The most frequent side effects reported with HEMGENIX were:
Liver enzyme elevations
Blood enzyme elevations
No inhibitors to factor IX were reported during the clinical trial
Infusion-related reactions (IRRs) occurred in 33% of people, with the majority resolving within a week
Symptoms of IRRs may include tightness of chest, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure
Most patients had mild elevations in liver enzymes without any symptoms. Some patients were treated with steroids to bring the levels back down.
In clinical trials, people could still use factor IX replacement products if needed. Speak with your doctor about specific situations where factor IX may be needed.
Patient portrayal; HEMGENIX not intended for women
These are not the only side effects possible with HEMGENIX. For additional safety information, please refer to the full prescribing information for HEMGENIX.
*HEMGENIX was studied in people aged 18 years or older with moderately severe to severe hemophilia B, including people with controlled HIV or resolved hepatitis B or C.
What is HEMGENIX?
HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:
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.