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ROCTAVIAN logo
[ON SCREEN TEXT]
ROCTAVIAN logo
Meet ANDREW – MY ROCTAVIAN EXPERIENCE
ROCTAVIAN Clinical Study Participant
[ANDREW SPEAKING]
My name is Andrew. I live in Fort Collins, Colorado. And I was born with severe hemophilia A.
[ON SCREEN TEXT]
Andrew
[ANDREW SPEAKING]
My brother’s 20 months older than me and also has severe hemophilia A. It was great to have a partner, to have an ally in hemophilia. He also kind of paved the way for me, showed me, what life would look like as a hemophiliac. Hemophilia affected me in a lot of different ways. It limited some of the activities I could do, but it also made me who I am.
I experience breakthrough bleeds several times a year, maybe once a month.
I wore this cast, um, over my right ankle, because it was a target joint. And it clicked every time I walked. And so, I remember freshman year of high school, this click following me around the hallways.
Hemophilia as an adult has hemmed me in a little bit.
I am happily married almost 15 years.
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Kirsten
[KIRSTEN SPEAKING]
I’m Kirstin, Andrew’s wife. So, we met our sophomore year, dated throughout college. And got married right when we graduated.
[ANDREW SPEAKING]
I have three kids, a boy and two little girls. I just cherish them and love being a dad. We, we spend a lot of time outdoors—biking and playing soccer and just generally exploring.
[KIRSTEN SPEAKING]
Andrew is an amazing dad. He’s so engaged. He’s always doing activities with them, like playing football, kicking a soccer ball around. He’s just a fun dad. We do a lot of dance parties in our house. And our kids are as active and energetic as he is.
[ANDREW SPEAKING]
Preparing for, infusions or, or for trips caused a decent amount of anxiety for me. I was always aware of where the nearest hospital would be, what my, my factor level was at, how many shots I brought, did I remember needles?
I would spend a decent amount of time prophylactically treating, but then also, treating in response to issues or treating before big events
I first heard about ROCTAVIAN from my brother.
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CONSIDERING ROCTAVIAN
[ON SCREEN TEXT & NARRATOR]
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.
IMPORTANT SAFETY INFORMATION
Do 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).
Please see additional safety information throughout and at the end of this video
[ANDREW SPEAKING]
My brother initially really motivated me to move forward with ROCTAVIAN. I was excited, and he was, too, about what this could mean for our lives. And I remember us just daydreaming about what it could lead to in our treatment of hemophilia. I loved the idea of helping future generations of hemophiliacs.
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SUPER: 90 out of 112 people responded to ROCTAVIAN, stopped, and stayed off continuous prophylaxis.
Results are based on 112 people whose data were collected for at least 6 months before receiving ROCTAVIAN and compared with their data over the 3-year
follow-up period.
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 3-year follow-up period. Prophylaxis is defined as the ongoing use of Factor VIII or another treatment to prevent bleeds.
The 3-year follow-up period began 5 weeks or more after administration and consists of a median follow-up of 3 years with a range of 1.7 to 3.7 years.
Data were collected for 6 months, and those results were annualized.
[ANDREW SPEAKING]
To learn more about ROCTAVIAN, I talked to my doctor, I talked to my nurse, my, my family. I Googled. I spent a lotta time learning from anybody I could about this new treatment and, and what it could mean for me. The more I researched and the more I studied about gene therapy, the more I realized that ROCTAVIAN was going to be the next step in treatment for me.
[KIRSTEN SPEAKING]
Andrew and I had a lot of conversations before we chose to do gene therapy talk to your doctor, talk to your loved one. The hemophilia community is amazing, so talk to them and research and make a decision for yourself.
Andrew was doing prophylaxis shots, and so, we talked about the pros and cons. And so, it felt like all of those things added up to saying, ‘Yes!’
[ON SCREEN TEXT & NARRATOR]
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. 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.
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TAKING THE NEXT STEP WITH ROCTAVIAN
[ANDREW SPEAKING]
I was so excited when I learned I was eligible to receive ROCTAVIAN. We, we got the tests back, and I was so excited to move forward.
[ON SCREEN TEXT]
Andrew’s ROCTAVIAN story started with simple eligibility tests. Connect with your healthcare team to schedule yours today
[ANDREW SPEAKING]
Leading up to infusion day. I had done all my research and had a bunch of conversations, and, and I felt ready and really hopeful for what this could mean for my health.
[ON SCREEN TEXT]
ROCTAVIAN is indicated for adults only.
Infusion time: 2 to 5 hours or longer based on infusion rate, weight, and response. Your healthcare team will monitor you before, during, and right after your infusion
[ANDREW SPEAKING]
My relationship with my doctor was great. Through this, this whole process, I’ve gotten to understand what’s happening in my body. My doctor and I would nerd out together on the science, you know, gene therapy.
My nurse, Julie, has been a rock for me. She’s been such a wealth of information, a person for me to process with and learn with.
There were lifestyle considerations I had to make for a year. And, and it just took informing myself, talking to my doctor about them. And for me, I chose to say, ‘yes,’ even in light of the considerations.
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Things to consider before treatment
[ANDREW SPEAKING]
I received my one-time infusion of ROCTAVIAN in October of 2018
[ON SCREEN TEXT]
Andrew received his ROCTAVIAN infusion in October 2018.
During 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, lightheadedness (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.
LIFE AFTER ROCTAVIAN
[ANDREW SPEAKING]
Monitoring for my ROCTAVIAN gene therapy involved weekly visits to the hemophilia treatment center. And, and I would go to the treatment center and get an update on my factor level. We’d also get an update on, on where my liver enzymes were at, because they carefully monitor your liver health. And for me I could see the effect the treatment had on my body.
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Follow-ups are important to help you see when your body starts to make its own Factor VIII
[ANDREW SPEAKING]
Seeing my factor levels at those weekly appointments gave me the confidence that ROCTAVIAN gene therapy was working for me that was my experience, but everybody’s experiences may be different.
Depending on your risk factors, an improvement in Factor VIII levels may mean an increased possibility of unwanted blood clots (so called “thromboses,” 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.
Those weekly visits felt like a lot for me at first. For the first couple weeks, I remember thinking, how am I going to get used to this? And then very quickly, it became part of my routine. And, actually, I ended up really enjoying getting to see my factor levels rise, and getting to talk with my doctor and, and get the inside scoop on what ROCTAVIAN gene therapy was doing for me.
My doctor informed me of the potential need for steroids for my liver health. I felt confident that they were taking care of me as they monitored me every week.
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Based 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’s health. 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.
[ANDREW SPEAKING]
My body making its own Factor VIII means my body is doing what it was supposed to do. I would think about how my body was doing, what my factor levels were at, what my activity level could be at.
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Individual results may vary.
After taking ROCTAVIAN, your doctor will monitor your lab tests 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.
Individual results may vary.
[ANDREW SPEAKING]
After receiving ROCTAVIAN it wasn’t until about a month and a half in that my doctor told me I could go off of prophylaxis.
I remember that first week without a shot. It had been 31 years where I’d, I’d gotten a shot three times a week. And that first week with no shot, I would bump myself and I wouldn’t bleed. And so, that’s when I really started to notice that ROCTAVIAN had changed something for me. Now my body was producing its own Factor and my treatment regime was so much different than it had been for the rest of my life before that.
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Andrew 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.
[ANDREW SPEAKING]
Yeah, since infusion, I haven’t had any prophylactic or shots in response to bleeds. I don’t think about my factor level before activity. I don’t have to dose up before I do, do something. I can just go.
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Results are based on 112 people whose data were collected for at least 6 months before receiving ROCTAVIAN and compared with their data over the 3-year follow-up period.
Throughout the 3-year follow-up period, average infusions were reduced from 136 per year before ROCTAVIAN to 5 per year after ROCTAVIAN.
Data were collected for 6 months, and those results were annualized.
[ANDREW SPEAKING]
This is my experience. Everyone’s experience may be different.
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PROPHYLAXIS FREE FOR YEARS
Patients who did not respond or lost response to ROCTAVIAN treatment can return to prophylaxis.
[ANDREW SPEAKING]
ROCTAVIAN has enabled my body to make its own Factor VIII. And so, I’m able to get off of regular prophylaxis.
[ON SCREEN TEXT]
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.
[ANDREW SPEAKING]
I used to spend two or three hours a week thinking about treatment and planning and infusing myself.
[ON SCREEN TEXT]
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.
[ANDREW SPEAKING]
I don’t have to schedule shots. I don’t have to set reminders for the next infusion. Now I always have factor on hand, but it’s freed up my time to pursue other activities. It’s given me time back.
[ON SCREEN TEXT]
It’s freed up my time to pursue other activities. It’s given me time back
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.
[ANDREW SPEAKING]
Advice I’d give to someone considering ROCTAVIAN to think of their health as more than just maintaining the status quo.
[ON SCREEN TEXT]
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.
[ANDREW SPEAKING]
Think of where you could go, if you’re not thinking about infusions
[ON SCREEN TEXT]
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.
[ANDREW SPEAKING]
You’ve gotten so used to where your treatment is taking you. Maybe there’s somewhere else you can go.
[ON SCREEN TEXT]
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.
[KIRSTEN SPEAKING]
If I could describe our experience with ROCTAVIAN in one word, it would be…
[ANDREW SPEAKING]
…eye-opening. That was my experience.
I’m happy with my decision.
Think about your day-to-day. Are there any changes you’d wanna make? Think about the impact that severe hemophilia A has on your daily life.
It’s pretty cool to know that my body is making its own factor now with ROCTAVIAN gene therapy.
Move forward with making your own Factor VIII.
[ON SCREEN TEXT]
MOVE FORWARD WITH MAKING YOUR OWN FACTOR VIII
Individual Factor VIII levels produced and durability of levels reached can vary.
[ON SCREEN TEXT & NARRATOR]
Indication and Important Safety 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.
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 at ROCTAVIAN.com.
[ROCTAVIAN logo]
ROCTAVIAN® is a registered trademark of BioMarin Pharmaceutical Inc. © 2024 BioMarin Pharmaceutical Inc. All rights reserved. US-ROC-00463 10/24
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.