We get it; considering any new therapy comes with a lot of questions. Making sure you feel informed is our priority. Here are answers to some common questions we have been asked about ROCTAVIAN.
And if you have questions that aren’t addressed here, a BioMarin representative is ready to connect.
ROCTAVIAN was FDA approved in 2023, based on the longest and largest gene therapy clinical study in severe hemophilia A.* Results were based on 3 years of data–and data continue to be collected.
*At approval, ROCTAVIAN had the longest and largest Phase 3 clinical study.
134 people received ROCTAVIAN as part of the clinical study—the longest and largest gene therapy study in severe hemophilia A.*
Hear firsthand experiences from people like Andrew S., who was part of the ROCTAVIAN clinical study.
Yes, ROCTAVIAN gene therapy is given as a one-time infusion for people with severe hemophilia A. It is given at a medical facility by a healthcare professional who is familiar with ROCTAVIAN.
Your doctor will watch for any infusion-related reactions, which are symptoms during, or shortly after, the infusion ends. Tell your healthcare team 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.
Your one-time 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 your infusion, it’s important to stick to a regular follow-up schedule with your healthcare team to know how ROCTAVIAN is working. Your doctor will perform blood tests to check your liver health. This monitoring becomes less frequent over time:
Based on your liver test results, you may need to take corticosteroids or another medicine for a period of time (several months or longer) to help decrease liver enzyme levels, which may cause side effects while you receive them. Talk to your doctor about these side effects and what you need to do to improve and maintain your liver’s health.
Your doctor will monitor you weekly while on corticosteroids.
ROCTAVIAN is a one-time treatment. Currently, treatment with ROCTAVIAN means you cannot receive another gene therapy for hemophilia.
The most common side effects of ROCTAVIAN are:
There are also important warnings and precautions for ROCTAVIAN. Learn more about ROCTAVIAN safety.
Because ROCTAVIAN works in the liver, you cannot 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.
Making the decision to receive one-time gene therapy with 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 for severe hemophilia A, determine which eligibility screening is right for you, and begin the treatment process. Our Doctor Discussion Guide can help get the conversation started.
ROCTAVIAN is approved for adults with severe hemophilia A who do not have antibodies to the AAV5 virus, which is determined by a blood test.
ROCTAVIAN is not intended for administration in women or children.
BioMarin is committed to providing personalized education and product support and resources, from the start of treatment and beyond. Whether you’re trying to understand insurance and financial assistance options or locate convenient eligibility and follow-up testing sites, our one-to-one support team will work with you and your healthcare team. Connect with a representative now.
The one-time cost of ROCTAVIAN is covered by most insurance plans. And if you have questions about costs or coverage, we can help. Our BioMarin RareConnections™ program has a team of experts ready to help you understand the insurance process and gain access to ROCTAVIAN. Connect with our treatment support team now.
Watch stories from patients who were part of the ROCTAVIAN clinical trial
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.
Please see additional safety information in the Prescribing Information and Patient Information.
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.