How ROCTAVIAN works
See how a one-time infusion delivers a working Factor VIII gene into the body.
Andrew is a runner, teacher, and father who used Factor VIII prophylaxis regularly until he received his one-time infusion as part of the ROCTAVIAN gene therapy clinical study. He worked with his doctor to monitor his levels over the following weeks to see if he could stop treatment. Throughout a 3-year follow-up period, Andrew was able to make his own Factor VIII and stop and stay off continuous prophylaxis.*†
Response to ROCTAVIAN can vary. After your ROCTAVIAN infusion, your healthcare team will review your lab test results and talk to you about whether you can stop prophylaxis, whether you should start prophylaxis again, and whether and how you should treat for any surgeries, procedures, injuries, or bleeds. Temporary infusion-related reactions are possible during, or shortly after, treatment.
*ROCTAVIAN was studied in 112 people whose data were collected for at least 6 months before their infusion (rollover population) and 22 people who immediately received their infusion (directly enrolled population). In the rollover population, the average Factor VIII level was 34% with a range of 0 to 291.4 at Year 3. Results were measured using a one-stage test, which is one way to measure Factor VIII levels. Range shows the lowest and highest numbers in a list. Andrew was part of the directly enrolled population.
†Prophylaxis is defined as the ongoing use of Factor VIII or another treatment to prevent bleeds. ROCTAVIAN worked for 80% (90/112) of people in the rollover population and 68% (15/22) of people in the directly enrolled population throughout the follow-up period.
See how a one-time infusion delivers a working Factor VIII gene into the body.
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.