ROCTAVIAN is designed to deliver a working copy of a Factor VIII gene to liver cells to allow those cells to produce Factor VIII.
ROCTAVIAN is given as a single, one-time infusion into a vein at a medical facility by a healthcare professional.
Your doctor will watch for any infusion-related reactions, which are symptoms during, or shortly after, the infusion ends. Tell your doctor or nurse immediately if you experience any of the following symptoms or any other symptoms during or shortly after the infusion: hives or other rashes, itching, sneezing, coughing, difficulty breathing, runny nose, watery eyes, tingling throat, nausea (feeling sick), diarrhea, low blood pressure, rapid heartbeat, light-headedness (near-fainting), fever, chills, or shivering.
The most common side effects of ROCTAVIAN are:
There are also important warnings and precautions for ROCTAVIAN. Click below to learn more about ROCTAVIAN safety.
Your ROCTAVIAN infusion will take 2 to 5 hours or longer depending on the infusion rate, your weight (since that determines dosing), and how you respond to the infusion. During and after your infusion, you will be monitored for any possible side effects. Your healthcare provider will determine when you can go home (usually later the same day).
After receiving ROCTAVIAN, it’s important to follow a regular follow-up schedule with your healthcare team.
Click below for more details.
ROCTAVIAN is a one-time treatment. Currently, treatment with ROCTAVIAN means you cannot receive another gene therapy for hemophilia.
Because ROCTAVIAN works in the liver, you can’t drink alcohol for the first 12 months after treatment. Talk to your healthcare team about how much alcohol may be acceptable after the first 12 months.
BioMarin RareConnectionsTM has a team of experts ready to help you understand the insurance process and gain access to ROCTAVIAN.
Making the decision to receive ROCTAVIAN is an important one that should be shared with your healthcare team. They can help you understand the potential benefits and risks of ROCTAVIAN, determine which eligibility screening is right for you, and begin the treatment process.
ROCTAVIAN is not intended for administration in women or children.
BioMarin is committed to providing personalized education and product support and resources throughout your ROCTAVIAN treatment journey. From understanding insurance and financial assistance options to locating convenient laboratory options for eligibility and follow-up testing, our one-to-one support team will work with you and your healthcare team from the start.
Do not take ROCTAVIAN if you:
What is the most important information I should know about ROCTAVIAN?
ROCTAVIAN may cause serious side effects during the infusion and afterward:
What should I tell my doctor before I get ROCTAVIAN?
Talk to your doctor about the following:
What should I avoid after taking ROCTAVIAN?
What are the possible side effects of ROCTAVIAN?
What other information should I know before getting ROCTAVIAN?
Talk to your doctor about the potential risks and benefits of ROCTAVIAN. Whether a patient experiences a benefit or not, the risks discussed here and with your doctor still apply.
These are not all the possible side effects of ROCTAVIAN. Talk to your doctor for medical advice about side effects. You may report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100 or FDA at 1-800-FDA-1088.
What is ROCTAVIAN?
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. ROCTAVIAN uses a modified virus, called a vector, to deliver a working copy of the Factor VIII gene to liver cells to enable your body to produce clotting factor on its own, which helps the blood to clot and prevents or reduces the occurrence of bleeding. The modified virus does not contain viral DNA and does not cause disease in humans.