Hemgenix
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.
What is Hemgenix (etranacogene dezaparvovec)?
What is Hemgenix (etranacogene dezaparvovec) used for?
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
How Hemgenix (etranacogene dezaparvovec) works
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.
Drug facts
Side effects of Hemgenix (etranacogene dezaparvovec)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Higher liver function tests (42%)
- Higher creatine kinase levels (42%)
- Infusion reactions (e.g., dizziness; 33%)
- Headache (18%)
- Flu-like symptoms (14%)
- Feeling tired (12%)
- Feeling unwell or general discomfort (12%)
Less Common Side Effects
- Nausea
- Belly pain or discomfort
- Chest discomfort
- Chills
- Itchy eyes
- Fever
- Hives
- Fast heart rate
Hemgenix (etranacogene dezaparvovec) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious infusion reaction: chest tightness, trouble breathing, rash, flushing, shivering, lightheadedness
- Serious liver damage: feeling tired or weak, poor appetite, unexplained weight loss, dark urine, whites of the eyes and skin turning more yellow, upper right stomach pain
Pros and cons of Hemgenix (etranacogene dezaparvovec)
Pros
- The first and only gene therapy for hemophilia B
- Given as a single dose for one-time treatment
- Few interactions with other medications
Cons
- 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
Pharmacist tips for Hemgenix (etranacogene dezaparvovec)
- 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.
Common questions about Hemgenix (etranacogene dezaparvovec)
Hemgenix (etranacogene dezaparvovec) is made by CSL Behring, a biopharmaceutical company.
Results from clinical studies showed that some people who received Hemgenix (etranacogene dezaparvovec) had higher levels of factor IX activity beginning at 3 weeks after the start of treatment. Factor IX activity levels stayed higher at least 1.5 years of treatment. In addition, people who received Hemgenix (etranacogene dezaparvovec) overall had fewer bleeding episodes than people who received factor IX replacement. Talk with your provider if you have questions about how well Hemgenix (etranacogene dezaparvovec) works.
Studies show that some people who received Hemgenix (etranacogene dezaparvovec) had higher levels of factor IX activity for at least 1.5 years during treatment. Keep in mind that different people might have different results with Hemgenix (etranacogene dezaparvovec). Ask your provider about what follow-up appointments or labs you might need after Hemgenix (etranacogene dezaparvovec) treatment to check whether this medication is working.
After you receive Hemgenix (etranacogene dezaparvovec), your provider will follow up with you for symptoms of bleeding. In addition, they'll regularly check your factor IX activity levels to see how well the medication is working. Since Hemgenix (etranacogene dezaparvovec) can harm your liver, your provider will also check your liver protein levels weekly for at least 3 months. If you're at higher risk for liver problems, you might need ultrasound screenings and other blood tests to check your liver health for at least 5 years. After you've received Hemgenix (etranacogene dezaparvovec), make sure to get your routine blood tests or lab work done on time. This helps your provider evaluate how well it's working and how safe it is for you.
Before you receive Hemgenix (etranacogene dezaparvovec), your provider will test your blood for factor IX inhibitors. These inhibitors are antibodies (proteins) that your immune system makes as a result of using common hemophilia B treatments like factor IX replacement products. The antibodies block factor IX from working in your body. Since Hemgenix (etranacogene dezaparvovec) helps your body make more factor IX, the medication won't work well if you have these inhibitors. If your lab results show that you have factor IX inhibitors, provider will discuss next steps or other treatment options that might work better for you.
How to save using GoodRx
Risks and warnings for Hemgenix (etranacogene dezaparvovec)
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.
Infusion reactions
- 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.
Liver damage
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.
Possible risk of liver cancer
- 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.
Risk of antibodies against Hemgenix
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.
Hemgenix (etranacogene dezaparvovec) dosage
Typical dosage for Hemgenix (etranacogene dezaparvovec)
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.
