Hemgenix (etranacogene dezaparvovec or etranacogene dezaparvovec-drlb) is a gene therapy that treats hemophilia B in adults. This medication can prevent or lower the number of bleeding episodes by helping the body make an important blood-clotting protein. It's given as a one-time intravenous (IV) infusion through the veins. Some common side effects of Hemgenix (etranacogene dezaparvovec) include dizziness, headache, and flu symptoms.
Treatment of hemophilia B in adults who:
Currently use factor IX replacement therapy, or
Have a history of life-threatening bleeding, or
Have repeated episodes of serious bleeding
Hemophilia B is a genetic condition. People with hemophilia B are missing or have low levels of factor IX, one of the clotting factors in the body. As a result, they're at higher risk for bleeding.
Hemgenix (etranacogene dezaparvovec) is a type of gene therapy. It uses viral vectors to deliver genes for factor IX into your liver. Once inside, your body uses these genes to make more copies of factor IX. Having more factor IX means that your body is better able to stop bleeds.
Source: DailyMed
The first and only gene therapy for hemophilia B
Given as a single dose for one-time treatment
Few interactions with other medications
Risk of liver problems
Commonly causes infusion reactions, such as dizziness
Not intended for females with hemophilia B; this medication wasn't studied in females
Make sure to get your blood tests or lab work done on time before and after receiving Hemgenix (etranacogene dezaparvovec). Doing so helps your provider make sure this medication is safe and will work well for you.
Consider planning for some extra time on the day of the Hemgenix (etranacogene dezaparvovec) infusion. How long the infusion takes will depend on how many infusion bags of medication you're receiving. Each bag is infused through the veins for about 1 hour. You might receive more than one bag to complete treatment.
Your healthcare provider will watch for infusion reactions during your infusion and for at least 3 hours after you've received Hemgenix (etranacogene dezaparvovec). This is because the medication might cause infusion reactions that are sometimes life-threatening. Tell your provider right away if you experience chest tightness, rash, flushing, shivering, or belly pain.
After receiving Hemgenix (etranacogene dezaparvovec), don't donate blood, organs, tissues, or cells at any point in the future. There might be a small amount of medication that stays in the body or bodily fluids for a long period of time.
If you experience excessive bleeding or frequent bleeding episodes after receiving Hemgenix (etranacogene dezaparvovec), tell your provider right away to get treated. They might also assess what other options are available for you.
Hemgenix (etranacogene dezaparvovec) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Risk factor: Fast infusion
Hemgenix (etranacogene dezaparvovec) can cause infusion reactions, such as rash, headaches, belly pain, lightheadedness, shivering, and flushing. Serious reactions like anaphylaxis or closing of the throat are possible as well. Your provider will watch for these reactions during your infusion and for at least 3 hours after you've received Hemgenix (etranacogene dezaparvovec). If you experience any reaction to Hemgenix (etranacogene dezaparvovec) during treatment, tell your provider right away so they can slow or stop your infusion until your symptoms go away. If your symptoms worsen, your provider might give you steroids or antihistamines for symptom relief.
Hemgenix (etranacogene dezaparvovec) can sometimes cause liver damage. This is most common in the first 3 or 4 months after the infusion, but it can happen later and last up to 2 years or more. You’ll need to get weekly blood tests done for at least the first 3 months after receiving Hemgenix (etranacogene dezaparvovec). This is so your provider can check your liver health. If you experience signs and symptoms of liver damage, such as upper right stomach pain, yellowing of the skin or whites of the eyes, or dark urine, talk with your provider immediately.
Risk factors: Older age | History of liver problems | Long-term use of alcohol
Based on the way Hemgenix (etranacogene dezaparvovec) works, there's a possible risk of developing liver cancer after treatment. Your risk might be higher if you have a history of liver problems, are older in age, or have a history of long-term alcohol use. For your safety, your provider might order ultrasound screenings and blood tests every year for at least 5 years after treatment to check for cancer. Speak with your provider if you have concerns about the risk of liver cancer. Let your provider know right away if you have symptoms of liver cancer, such as swelling in the belly, yellowing of the skin or whites of the eyes, or confusion.
When you receive Hemgenix (etranacogene dezaparvovec), there's a risk that your body will make antibodies against parts of Hemgenix (etranacogene dezaparvovec). If you have antibodies against Hemgenix (etranacogene dezaparvovec), this can affect how the medication works. Before starting this medication, it's recommended that you call the manufacturer of Hemgenix (etranacogene dezaparvovec) (CSL Behring) at 1-800-504-5434 or speak with your provider regarding possible antibody testing through clinical studies.
Each milliliter (mL) of Hemgenix contains 1 × 1013 genomic copies (gc) of the working factor IX gene. Your provider will determine the dose of Hemgenix (etranacogene dezaparvovec) based on your bodyweight.
The typical recommended dose is 2 mL/kg of body weight injected through the veins as a one-time infusion by a healthcare provider.
Treatment of hemophilia B in adults who:
Currently use factor IX replacement therapy, or
Have a history of life-threatening bleeding, or
Have repeated episodes of serious bleeding
To prevent or treat bleeding in adults and children with hemophilia B
To prevent or treat bleeding in adults with hemophilia B
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