{"id":6340,"date":"2024-10-02T19:24:41","date_gmt":"2024-10-02T19:24:41","guid":{"rendered":"https:\/\/www.roctavian.com\/en-us\/?page_id=6340"},"modified":"2025-12-17T15:57:30","modified_gmt":"2025-12-17T15:57:30","slug":"andrew-w","status":"publish","type":"page","link":"https:\/\/www.roctavian.com\/en-us\/roctavian-stories\/andrew-w\/","title":{"rendered":"ANDREW-W"},"content":{"rendered":"
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Andrew W. Transcript<\/h1>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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[ON SCREEN TEXT]<\/p>\n

ROCTAVIAN logo<\/p>\n

[NARRATOR AND ON SCREEN TEXT]
\nWhat is ROCTAVIAN?
\nROCTAVIAN 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.<\/p>\n

[ON SCREEN TEXT]
\nMy ROCTAVIAN Experience
\nMeet Andrew
\nROCTAVIAN clinical study participant<\/p>\n

[ANDREW W.]
\nHi, I’m Andrew. I live with severe hemophilia A, and this is my ROCTAVIAN story.<\/p>\n

[ON SCREEN TEXT]
\nIMPORTANT SAFETY INFORMATION<\/p>\n

[NARRATOR AND ON SCREEN TEXT]
\nDo not take ROCTAVIAN if you have an active infection or if you have a long-term infection that is not controlled by the medicines you take, have scarring of the liver (significant liver fibrosis or cirrhosis), are allergic to mannitol (an inactive ingredient in ROCTAVIAN).<\/p>\n

Please see additional safety information throughout and at the end of this video.<\/p>\n

[ON SCREEN TEXT]
\nAndrew W., received ROCTAVIAN in 2018 as part of a clinical trial<\/p>\n

[ANDREW W.]
\nI\u2019m from Louisville, Kentucky, where I live with my wife, my daughter, and my niece and nephew.<\/p>\n

[ON SCREEN TEXT]
\nWhy I chose ROCTAVIAN<\/p>\n

[ANDREW W.]
\nI first heard about ROCTAVIAN from my nurse at my hemophilia treatment center. What really got me excited about ROCTAVIAN when I started doing more research and talking to more doctors about it was just that possibility that I could produce Factor VIII. My doctor and I discussed the possibility and my doctor and I discussed the risks and benefits of it, as well as some of the lifestyle changes that was going to have to come with being a part of the clinical trial.<\/p>\n

[ON SCREEN TEXT]
\nROCTAVIAN 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. Your doctor may perform regular monitoring if you have pre-existing risk factors for developing liver cancer. In the event of cancer, your doctor may send a sample to BioMarin Pharmaceutical Inc. for further testing.<\/p>\n

ROCTAVIAN gene therapy starts with simple eligibility tests. Talk to your healthcare team about eligibility testing for ROCTAVIAN gene therapy.<\/p>\n

[ANDREW W.]
\nAfter I got a phone call from somebody in my care team explaining that I was eligible for this, everybody was really excited and thrilled that we could move forward.<\/p>\n

I never imagined a day where I wouldn\u2019t have to actively treat with factor.<\/p>\n

[ON SCREEN TEXT]
\n5 years after ROCTAVIAN, more than 70% of people who had responded to ROCTAVIAN stopped\u2014and stayed off\u2014 continuous prophylaxis<\/p>\n

ROCTAVIAN was studied in 112 people whose data was collected for at least 6 months before their infusion (rollover population) and 22 people who immediately received their infusion (directly enrolled population).<\/p>\n

ROCTAVIAN worked for 71% (80\/112) of people in the rollover population and 59% (13\/22) of people in the directly enrolled population throughout the 5-year follow-up period.<\/p>\n

Prophylaxis is defined as the ongoing use of Factor VIII or another treatment to prevent bleeds.<\/p>\n

The follow-up period began 5 weeks or more after administration and consists of a median follow-up of 5 years with a range of 1.7 to 5.2 years.<\/p>\n

[Infographic of 70%\u201480 of 112 people]<\/p>\n

[ON SCREEN TEXT]
\nSafety profile of ROCTAVIAN after 5 years<\/p>\n

The safety of ROCTAVIAN is being evaluated in a clinical trial<\/p>\n