Roctavian
Roctavian (valoctocogene roxaparvovec or valoctocogene roxaparvovec-rvox) is a gene therapy that treats severe hemophilia A in adults. This medication can prevent or lower the number of bleeding episodes by helping the body make an important blood-clotting protein. Roctavian (valoctocogene roxaparvovec) is given as a one-time intravenous (IV) infusion through the veins by a healthcare provider. Common side effects of this medication include nausea, tiredness, and infusion-related reactions (e.g., rash and itching).
What is Roctavian (valoctocogene roxaparvovec)?
What is Roctavian (valoctocogene roxaparvovec) used for?
- Treatment of severe hemophilia A in adults
How Roctavian (valoctocogene roxaparvovec) works
Hemophilia A is a genetic condition where people have low levels of factor VIII, one of the clotting factors. As a result, they're at higher risk for bleeding.
Roctavian (valoctocogene roxaparvovec) is a type of gene therapy. It uses empty viral vectors to deliver working factor VIII genes into your body. Then, your body uses the genes to make more factor VIII. Having more factor VIII means that your body is better able to stop bleeds.
Drug facts
Side effects of Roctavian (valoctocogene roxaparvovec)
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 (up to 81%)
- Higher creatine kinase levels (45%)
- Nausea (31%)
- Feeling tired (16%)
- Headache (7%)
- Infusion-related reactions (7%)
- Vomiting (6%)
- Belly pain (6%)
Less Common Side Effects
- Stomach flu
- Rash
- Diarrhea
- Dizziness
Roctavian (valoctocogene roxaparvovec) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious infusion reaction: hives, itching, rash, sneezing, coughing, shortness of breath, runny nose, watery eyes, tingling throat, nausea, diarrhea, fast heart rate, fever, chills
- Blood clots: dizziness, confusion, chest pain, sweating, nausea, trouble breathing, swelling or pain in the legs, weakness (especially only on one side of the body)
- 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
The following Roctavian (valoctocogene roxaparvovec) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Blood clot—pain, swelling, or warmth in the leg, shortness of breath, chest pain
- Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
- Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Roctavian (valoctocogene roxaparvovec)
Pros
- Given as a single dose for one-time treatment
- Shown to lower bleeding episodes for up to 3 years
- The first gene therapy for severe hemophilia A
Cons
- Risk of liver problems
- Can cause infusion-related reactions, such as rash and hives
- Not intended for females with hemophilia A; this medication wasn't studied in females
Pharmacist tips for Roctavian (valoctocogene roxaparvovec)
Tips for before the Roctavian (valoctocogene roxaparvovec) infusion:
- Before you receive Roctavian (valoctocogene roxaparvovec), your provider will order blood tests and lab tests to make sure this medication is safe for you. For example, they'll need to check if you've antibodies against the viral vector (the "truck" that delivers the medication to you) or if you have factor VIII inhibitors. Be sure you go to your lab appointments so that your provider can talk with you about next-steps.
Tips for the Roctavian (valoctocogene roxaparvovec) infusion day:
- Plan extra time on the day of the Roctavian (valoctocogene roxaparvovec) infusion. How long the infusion takes will depend on how many infusion bags of medication you're receiving. Typically, it takes 2 to 5 hours (sometimes longer) to complete the infusion. Dress comfortably and consider bringing books or music to help you pass the time.
- Your healthcare provider will watch for infusion reactions during your infusion and for at least 3 hours after you've received Roctavian (valoctocogene roxaparvovec). This is because the medication might cause infusion reactions that are sometimes life-threatening. Tell your provider right away if you experience hives, itching, rash, and trouble breathing.
Tips for after the Roctavian (valoctocogene roxaparvovec) infusion:
- Your provider will order labs for you weekly after your Roctavian (valoctocogene roxaparvovec) infusion, at least for the first year. This is to check how well Roctavian (valoctocogene roxaparvovec) is working for you and to check your liver health.
- After receiving Roctavian (valoctocogene roxaparvovec), don't donate blood, organs, tissues, semen, 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 serious bleeding or bleeding episodes often after receiving Roctavian (valoctocogene roxaparvovec), tell your provider right away to get treated. They might order lab work to see how well Roctavian (valoctocogene roxaparvovec) is working for you. They might also assess what other options are available for you.
- Roctavian (valoctocogene roxaparvovec) can raise your risk of blood clots. If you experience symptoms of a blood clot, such as a stroke (e.g., weakness or numbness on one side of the body, trouble speaking, blurry vision, dizziness) or a heart attack (e.g., chest pain, sweating, nausea, or dizziness), get medical attention right away.
- Make sure to use condoms or other contraceptives for at least 6 months after receiving Roctavian (valoctocogene roxaparvovec). If your partner is able to have children, it's important for them to avoid pregnancy during this time. The viral vector can stay in the testes for up to 6 months after the dose; it's unclear how this might affect a developing baby.
Common questions about Roctavian (valoctocogene roxaparvovec)
Roctavian (valoctocogene roxaparvovec) helps your body make more factor VIII proteins, which can lower the risk of excessive bleeding. Studies show that people who received Roctavian (valoctocogene roxaparvovec) had fewer serious bleeding episodes for 3 years after treatment than those who didn't. In addition, most people were able to stay off factor VIII replacement therapy after receiving Roctavian (valoctocogene roxaparvovec). Keep in mind that Roctavian (valoctocogene roxaparvovec) might not be right for everyone. Talk with your provider about the best treatment plan for you.
No, Roctavian (valoctocogene roxaparvovec) isn't a cure for hemophilia A. Currently, all available treatments or medications for hemophilia A only temporarily replace the missing factor VIII (blood clotting factor) so that your blood can clot normally. People with hemophilia A should regularly watch out for bleeding symptoms ranging from nosebleeds, bleeding from the gums to excessive bruising or bleeding after an injury. Make sure to let your provider know if you've an upcoming surgery or dental procedure so they can help you lower your risk of major bleeds.
You should avoid drinking alcohol for at least a year after receiving Roctavian (valoctocogene roxaparvovec). This is Roctavian (valoctocogene roxaparvovec) can cause liver problems, and drinking alcohol can worsen this side effect. Depending on your liver health, your provider will let you know what's a safer amount to drink after the first year.
After you receive Roctavian (valoctocogene roxaparvovec), your provider will order blood tests weekly to check your factor VIII activity levels to see how well the medication is working. At the same time, they'll also check your liver labs to see if you have any liver irritation or damage, which can be a side effect of Roctavian (valoctocogene roxaparvovec). In addition, your provider will check for factor VIII inhibitors, which are antibodies your body might make against factor VIII; these antibodies can prevent this medication from working well. Your provider will also be doing liver ultrasounds yearly to check for symptoms of liver cancer, which is also a possible side effect of Roctavian (valoctocogene roxaparvovec).
Before you receive Roctavian (valoctocogene roxaparvovec), your provider will test your blood for factor VIII inhibitors. These inhibitors are antibodies (proteins) that your immune system makes as a result of using common hemophilia A treatments like factor VIII replacement products. The antibodies block factor VIII from working in your body. Since Roctavian (valoctocogene roxaparvovec) helps your body make more factor VIII, the medication won't work well if you have these inhibitors. Your provider will discuss other options that might work better for you.
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Risks and warnings for Roctavian (valoctocogene roxaparvovec)
Roctavian (valoctocogene roxaparvovec) 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
Roctavian (valoctocogene roxaparvovec) can cause infusion reactions, such as rash, itching, sneezing, coughing, fever, nausea, diarrhea, and more. 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 Roctavian (valoctocogene roxaparvovec). If you experience any reaction to Roctavian (valoctocogene roxaparvovec) 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
- Risk factors: History of liver problems | Long-term or excessive use of alcohol
Roctavian (valoctocogene roxaparvovec) can cause liver problems, typically within the first year of treatment. Many people who develop liver problems don't notice symptoms, but do see higher liver enzymes through labs. To check for liver problems, your provider order blood tests weekly after you've received Roctavian (valoctocogene roxaparvovec) for at least the first 6 months. Avoid alcohol for at least a year after infusion to prevent liver damage. 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 Roctavian (valoctocogene roxaparvovec) 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.
Possible risk of blood clot
- Risk factors: Higher factor VIII levels | History of heart problems | History of blood clot events
Rarely, Roctavian (valoctocogene roxaparvovec) can cause serious blood clots since it raises the levels of the blood-clotting protein, factor VIII, in your body. Blood clots can be life-threatening and can happen anywhere in your body. Get medical attention right away if you have any signs or symptoms of a blood clot:
- Brain: weakness or numbness in your arms, legs, or face on one side of the body, trouble speaking, blurry vision, dizziness, loss of balance, trouble walking, or confusion - Eyes: trouble seeing, bulging eye, double vision - Heart: chest pain, sweating, nausea, or dizziness - Lungs: trouble breathing, chest pain, or cough - Legs: cramps, swelling, pain, or muscle tightness
Your risk of getting a blood clot is higher if you have certain heart conditions. Talk with your provider if you have any questions regarding your risk of blood clots.
Roctavian (valoctocogene roxaparvovec) dosage
Typical dosage for Roctavian (valoctocogene roxaparvovec)
Each milliliter (mL) of Roctavian (valoctocogene roxaparvovec) contains 2 × 1013 vector copies (vc) of the working factor VIII gene. Your provider will determine the dose of Roctavian (valoctocogene roxaparvovec) based on your body weight.
The typical recommended dose is 3 mL/kg of body weight injected through the veins by a healthcare provider as a one-time infusion.
Roctavian (valoctocogene roxaparvovec) contraindications
Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Roctavian (valoctocogene roxaparvovec) will not be safe for you to take.
- Active, chronic (long-term), or recurrent infection
- Liver fibrosis
- Cirrhosis (liver scarring)
- Allergic reaction to Bronchitol (mannitol)
