Working gene DNA is made in a lab and contains instructions for the body to make the Factor VIII proteins that are missing or not working right in people with hemophilia A
The shell of a type of nonpathogenic virus found throughout the world acts like a transport vehicle, protecting and delivering the working gene DNA to where it needs to go in the body. Only working gene DNA is inside the shell, and there is no viral DNA
Scientists create the working gene DNA and viral shell together to form a therapeutic vector. This is ROCTAVIAN
While some viruses can make you sick, others can be used to treat illnesses, deliver vaccines, and even fight against infections. The viral shell used in ROCTAVIAN was chosen for many reasons, like size and the way it can travel to certain parts of the body. In our case, the viral shell travels to the liver where Factor VIII can be made.
During and after your infusion your doctor will monitor you for infusion-related reactions.
ROCTAVIAN can insert itself into the DNA of human body cells. The effect that insertion may have on those cells is unknown, but such events may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with ROCTAVIAN. Learn more here.
Find out how people responded to a one-time infusion of ROCTAVIAN.
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.