{"id":2133,"date":"2023-05-29T21:56:40","date_gmt":"2023-05-29T21:56:40","guid":{"rendered":"https:\/\/www.roctavian.com\/en-us\/?page_id=2133"},"modified":"2025-12-15T13:32:37","modified_gmt":"2025-12-15T13:32:37","slug":"faqs","status":"publish","type":"page","link":"https:\/\/www.roctavian.com\/en-us\/faqs\/","title":{"rendered":"FAQs"},"content":{"rendered":"
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FREQUENTLY ASKED QUESTIONS\n<\/h1>\n \t\t\t\t\t<\/div>\t\t\n
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If you have questions that aren\u2019t answered here, we can help.<\/p>\n <\/div>\n

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CONNECT WITH US<\/a><\/p>\n <\/div>\n <\/div>\n <\/div>\n

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Steven S. received ROCTAVIAN in 2019 as part of a clinical study.<\/p>\n <\/div>\n\n

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ROCTAVIAN is not intended for administration in women.<\/p>\n <\/div>\n \t<\/div>\n<\/div>\n

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Questions we\u2019ve been asked by the hemophilia community<\/p>\n <\/div>\n <\/div>\n <\/div>\n\n\n

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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.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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What is ROCTAVIAN used for?\n<\/h2>\n <\/div>\n
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ROCTAVIAN is a one-time treatment for adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test.
\nROCTAVIAN was FDA approved in 2023, based on the longest and largest gene therapy clinical study for severe hemophilia A.* The safety profile and efficacy of ROCTAVIAN have been studied for 5 years\u2014and data continue to be collected.<\/p>\n

*At FDA approval, ROCTAVIAN had the longest and largest Phase 3 clinical study for gene therapy in severe hemophilia A.<\/small><\/p>\n <\/div> <\/div>\n <\/div>\n

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How does ROCTAVIAN work?\n<\/h2>\n <\/div>\n
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ROCTAVIAN is one-time gene therapy. ROCTAVIAN uses a modified virus, called a vector, to deliver a working copy of the Factor VIII gene to liver cells. This helps your body produce clotting factor on its own. The modified virus does not contain viral DNA and does not cause disease in humans.<\/p>\n <\/div> <\/div>\n <\/div>\n

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How many people have received ROCTAVIAN?\n<\/h2>\n <\/div>\n
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134 people received ROCTAVIAN as part of the clinical study\u2014the longest and largest gene therapy study
\nfor severe hemophilia A.*<\/p>\n

Hear firsthand experiences<\/a> from people who chose ROCTAVIAN.<\/p>\n

*At FDA approval, ROCTAVIAN had the longest and largest Phase 3 clinical study for gene therapy in severe hemophilia A.<\/small><\/p>\n <\/div> <\/div>\n <\/div>\n

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What is the recommended dosage?\n<\/h2>\n <\/div>\n
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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.<\/p>\n

During the infusion, 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.<\/p>\n <\/div> <\/div>\n <\/div>\n

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How long will my infusion take?\n<\/h2>\n <\/div>\n
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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).<\/p>\n <\/div> <\/div>\n <\/div>\n

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How often will I need to meet with my doctor after ROCTAVIAN?\n<\/h2>\n <\/div>\n
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After your infusion, it\u2019s 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:<\/p>\n