{"id":3584,"date":"2025-11-17T12:50:50","date_gmt":"2025-11-17T12:50:50","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?page_id=3584"},"modified":"2025-11-25T16:55:35","modified_gmt":"2025-11-25T16:55:35","slug":"dave-n","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/dave-n\/","title":{"rendered":"dave-N"},"content":{"rendered":"
\n
\n
\n \n
\n

Dave’s Story<\/h1>\n

Video Transcript<\/h1>\n

[ON SCREEN TEXT]
\nROCTAVIAN logo
\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

[DAVE SPEAKING]
\nMy name is Dave, and this is my severe hemophilia A story.<\/p>\n

[ON SCREEN TEXT]
\nMeet Dave
\nMy ROCTAVIAN experience
\nReceived ROCTAVIAN in 2024<\/p>\n

[DAVE SPEAKING]
\nI\u2019m 39 years old.<\/p>\n

Growing up with severe hemophilia A was challenging. I would say that there was never really an easy time.<\/p>\n

[ON SCREEN TEXT]
\nDave<\/p>\n

[DAVE SPEAKING]
\nI didn\u2019t participate in any sports\u2014official sports that the school sponsored\u2014just because it was too difficult to heal my injuries with the medicine that was available at the time.<\/p>\n

[ON SCREEN TEXT]
\nROCTAVIAN is indicated for adults only. ROCTAVIAN is not intended for administration in women.<\/p>\n

[DAVE SPEAKING]
\nAs an adult, I\u2019m doing more traveling. I have more activities. I\u2019m taking care of other people. So I have to make sure I really focus on taking care of myself so I\u2019m able to do those things.<\/p>\n

[ON SCREEN TEXT]
\nROCTAVIAN is indicated for adults only.<\/p>\n

[DAVE SPEAKING]
\nI have two kids, Ava and Anthony…<\/p>\n

[ON SCREEN TEXT]
\nJenna
\n[DAVE SPEAKING]
\n…and a wife, Jenna.<\/p>\n

[JENNA SPEAKING]
\nI\u2019m Jenna, and I\u2019m Dave\u2019s wife. We have been married for 2 years, and we met in high school.<\/p>\n

[DAVE SPEAKING]
\nIn my free time, I like to play video games, go kayaking. We like to go fishing and just try to stay active. So I always have to think about hemophilia and how it affects me and how I need to plan my life kind of around it.<\/p>\n

So prior to ROCTAVIAN, I was okay with the prophylaxis that I was on, but I was never fully comfortable with it. Even treating once a month, I dreaded that next treatment.<\/p>\n

[JENNA SPEAKING]
\nI had to help Dave remember to treat some days.<\/p>\n

[ON SCREEN TEXT]
\nConsidering ROCTAVIAN<\/p>\n

[ON SCREEN TEXT & NARRATOR]
\nWhat 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.<\/p>\n

IMPORTANT SAFETY INFORMATION
\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), or 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

[DAVE SPEAKING]
\nSo I first heard about ROCTAVIAN gene therapy from my doctor at my yearly visit. And I was on board from the second he started telling me about it. I always wanted to get off treating on demand, prophylaxis. So, when the option came, I jumped at it.<\/p>\n

[ON SCREEN TEXT]
\nROCTAVIAN did not work for everyone. Some patients did not respond to treatment or lost response to treatment.<\/p>\n

[DAVE SPEAKING]
\nThe steps that I took to learn more about ROCTAVIAN were just talking to my doctors.<\/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

[DAVE SPEAKING]
\nMy doctor discussed with me the risks and benefits as well as the lifestyle changes that come with ROCTAVIAN.<\/p>\n

[ON SCREEN TEXT]
\nThings to consider before treatment
\n\u2022 Your healthcare team will ask you to not drink alcohol for the first 12 months after treatment
\n\u2022 Check with them before taking any medicines, herbal products, or supplements
\n\u2022 Use an effective form of birth control with your female partner for 6 months after ROCTAVIAN
\n\u2022 Do not donate blood, organs, tissues, or cells for transplantation<\/p>\n

[DAVE SPEAKING]
\nMy biggest concern about taking ROCTAVIAN is that if it didn\u2019t give me the results that I was looking for, that I would have to go back to being on prophylaxis.<\/p>\n

[ON SCREEN TEXT]
\nDave\u2019s ROCTAVIAN story started with simple eligibility tests. Connect with your healthcare team to schedule yours today.<\/p>\n

[DAVE SPEAKING]
\nThe eligibility process for me was luckily very easy.
\nAnd then I made sure that my liver was healthy and that I didn\u2019t contain any of the antibodies that ROCTAVIAN worked with to make sure that my body wouldn\u2019t fight it when I received it.<\/p>\n

[ON SCREEN TEXT]
\nDave worked with his doctor to stop prophylaxis in the weeks following his ROCTAVIAN infusion. 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.<\/p>\n

Depending on your risk factors, an improvement in Factor VIII levels may mean an increased possibility of unwanted blood clots (so called \u201cthromboses,\u201d in either veins or arteries). You and your doctor should discuss your risk factors before and after treatment and how to recognize symptoms of unwanted clots and what to do if you think you may have one. No blood clots or thromboembolic events were observed in ROCTAVIAN clinical trials.<\/p>\n

Infusion Day<\/p>\n

[DAVE SPEAKING]
\nI would describe my infusion day by just saying, \u201cExciting!\u201d It was a very early morning because it\u2019s a very long day of getting the infusion. All I had to do was sit down and relax, and the center took care of everything for me.<\/p>\n

[ON SCREEN TEXT & NARRATOR]
\nDuring and in the hours following the infusion, tell your doctor or nurse immediately about any symptoms you experience, including 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. Talk to your doctor about what to do if you experience any side effects after you leave the infusion.<\/p>\n

[ON SCREEN TEXT]
\nFollow-ups are important to check your liver health and help you see when your body starts to make its own Factor VIII
\nFor the first 6 months after your infusion, you\u2019ll need weekly blood tests to check your liver health and measure Factor VIII levels
\nFollow -ups will become less frequent over time and help your healthcare team know when you can stop routine prophylaxis<\/p>\n

[DAVE SPEAKING]
\nFollow-up post-infusion, I had weekly blood tests to make sure that all the levels of my liver were looking good and healthy.<\/p>\n

[ON SCREEN TEXT]
\nResponse 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.<\/p>\n

[DAVE SPEAKING]
\nSo having the weekly blood test and knowing what my factor levels were was very exciting for me.<\/p>\n

[ON SCREEN TEXT]
\nBased 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\u2019s health. Your follow-up schedule may need to change if you are taking corticosteroids.<\/p>\n

Most patients in the clinical trial required corticosteroids and the median duration of corticosteroid use was 8 months with a range of 3 weeks to over 2 years.<\/p>\n

[DAVE SPEAKING]
\nAnd that went on for about 6 months, and then I went to every other week for the following 6 months.<\/p>\n

I did take steroids to help my liver kind of control the changes that were going on.<\/p>\n

[ON SCREEN TEXT]
\nLife after ROCTAVIAN<\/p>\n

After treatment with ROCTAVIAN, you will be asked to enroll in a 15-year registry to help study the long-term safety of the treatment and how well it continues to work.
\n[DAVE SPEAKING]
\nROCTAVIAN has impacted my treatment routine because I don\u2019t have a routine anymore.<\/p>\n

[ON SCREEN TEXT]
\nDave worked with his doctor to stop prophylaxis in the weeks following his ROCTAVIAN infusion. 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.<\/p>\n

[DAVE SPEAKING]
\nAfter my ROCTAVIAN infusion, I was on prophylaxis for a month until my doctor told me I didn\u2019t have to do prophylaxis anymore.<\/p>\n

[ON SCREEN TEXT]
\n5 years after ROCTAVIAN, more than 70% of people (80 out of 112) had responded then stopped\u2014and stayed off\u2014continuous prophylaxis*\u2020<\/p>\n

Some people did not respond to treatment or lost response to treatment. In clinical studies, these people were able to resume prophylaxis. Your healthcare team will talk with you to decide what\u2019s right for you.<\/p>\n

*Data were collected for 6 months, and those results were annualized.
\n\u2020ROCTAVIAN 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

[DAVE SPEAKING]
\nAfter ROCTAVIAN, my factor levels began to rise and peaked after a year.
\nEverybody has different results.<\/p>\n

[ON SCREEN TEXT]
\nNausea 32.1% of people
\nFatigue 15.7% of people
\nHeadache 7.5% of people
\nInfusion-related reactions 9% of people
\nVomiting 6% of people
\nAbdominal pain 6% of people
\nElevated liver enzyme levels that were temporary and asymptomatic
\n82.8% of people experienced ALT levels above the upper limit of normal<\/p>\n

ALT=alanine aminotransferase.<\/p>\n

[NARRATOR SPEAKING]
\nIn the clinical study, the most common side effects were nausea, fatigue, headache, infusion-related reactions, vomiting, abdominal pain, and elevated liver enzyme levels.<\/p>\n

[ON SCREEN TEXT]
\nResults are based on 112 people whose data were collected for at least 6 months before their infusion (rollover population) and compared with their data over the 5-year follow-up period.<\/p>\n

Before ROCTAVIAN*\u2020
\nPatients averaged 136 infusions per year<\/p>\n

95% Infusion reduction through 5 years (n=112)<\/p>\n

After ROCTAVIAN*
\nInfusions declined to an average of 7 per year<\/p>\n

Throughout the 5-year follow-up period, average infusions were reduced from 136 per year before ROCTAVIAN to 7 per year after ROCTAVIAN.<\/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.
\n\u2020Data were collected for 6 months, and those results were annualized.<\/p>\n

[DAVE SPEAKING]
\nFor somebody who\u2019s considering ROCTAVIAN, I would say do your research. Make sure you have all the information.<\/p>\n

But for me, I would say go for it.<\/p>\n

[ON SCREEN TEXT]
\nWhile the majority of patients experience a benefit from ROCTAVIAN, the treatment response, duration, and side effects may vary. Some patients do not experience a benefit from ROCTAVIAN. It is not possible to predict if and how much a patient may benefit.<\/p>\n

[JENNA SPEAKING]
\nIt is quite a process, but it was nice to see, and just be supportive along the way no matter what the results are.<\/p>\n

[DAVE SPEAKING]
\nSo freedom from prophylaxis has changed our lives. ROCTAVIAN was the right choice for treating my severe hemophilia A. Talk to your doctor to see if ROCTAVIAN is the right choice for you.<\/p>\n

[ON SCREEN TEXT]
\nSpontaneity can be your focus
\nOne infusion. Years of freedom from continuous prophylaxis is possible.<\/p>\n

[ON SCREEN TEXT & NARRATOR]<\/p>\n

Indication and Important Safety Information<\/strong><\/p>\n

What is ROCTAVIAN?<\/strong><\/p>\n

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.<\/p>\n

Do not take ROCTAVIAN if you:<\/strong><\/p>\n