{"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":"2024-10-28T20:15:05","modified_gmt":"2024-10-28T20:15:05","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’s Story<\/h1>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Video transcript<\/h2>\n

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

MY ROCTAVIAN EXPERIENCE<\/p>\n

Meet Andrew
\nROCTAVIAN clinical study participant<\/p>\n

[ANDREW SPEAKING]<\/p>\n

Hi, I’m Andrew. I live with severe hemophilia A, and this is my ROCTAVIAN story.<\/p>\n

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

What is ROCTAVIAN?<\/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.<\/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), are allergic to mannitol (an inactive ingredient in ROCTAVIAN). Please see additional safety information throughout and at the end of this video.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I\u2019m from Louisville, Kentucky, where I live with my wife, my daughter, my niece and nephew. It\u2019s been about 3 years since I\u2019ve had my one-time ROCTAVIAN infusion.<\/p>\n

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

WHY I CHOSE ROCTAVIAN<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I first heard about ROCTAVIAN from my nurse at my hemophilia treatment center. My nurse saw this clinical trial opportunity come across and thought, you know, Andrew would be perfect for this. You’ve been waiting for a long time.<\/p>\n

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 actually could produce Factor VIII.<\/p>\n

And, I think the first person I called was actually, my dad. You know, \u2018Dad, they may have a, they may have found something\u2019 and we were both kinda crying, tearing up a little bit. And the second call though, was definitely my wife.<\/p>\n

We also were crying and a tearing up a little bit. I was like, \u2018This could be it\u2019. And then I tried to kind of reel it back in a little bit, you know, it may not work, maybe I should temper my expectations until we kind of see how it goes.<\/p>\n

My doctor and I discussed some of 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 & NARRATOR]<\/p>\n

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.<\/p>\n

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

[ANDREW SPEAKING]<\/p>\n

Eligibility is a big word, but as far as the blood test portion of it, I just went to my hemophilia treatment center and they just did a simple blood draw, and we just waited for the results.<\/p>\n

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

ROCTAVIAN gene therapy starts with simple eligibility tests.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

After 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

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

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

[ANDREW SPEAKING]<\/p>\n

Through this decision process, friends and family played a big role, but my care team did as well. You know, I’ve been with a lot of these doctors and nurses for almost all my life. So, you know, kind of bouncing ideas and questions off of them and getting their feedback was definitely a big part in my ultimate decision to move forward with it.<\/p>\n

When I was discussing ROCTAVIAN with my doctor and kind of my family, and kind of thinking back on all the hundreds and hundreds of infusions that I\u2019d had previously.<\/p>\n

I understand that there\u2019s risk with any clinical trial, but my thought process was at least I\u2019ve done the research. I want to do this. This is what I’ve been waiting for!<\/p>\n

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

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

[ROCTAVIAN logo]<\/p>\n

Over the 3-year follow-up period, 90 out of 112 people<\/p>\n

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.*<\/p>\n

90 out of 112 people responded to ROCTAVIAN and were able to stop and stay off continuous prophylaxis. 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).<\/p>\n

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.<\/p>\n

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

*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. Median is the middle number in a list of numbers arranged from smallest to largest.<\/p>\n

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

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

[ROCTAVIAN logo]<\/p>\n

ROCTAVIAN INFUSION DAY<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I remember everything about my infusion day, everything that happened that day.<\/p>\n

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

It took a few hours. They monitored me throughout and continued to check in on me afterwards.<\/p>\n

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

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.<\/p>\n

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

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.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

My doctor explained to me the lifestyle changes that I would have to make.<\/p>\n

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

For 6 months after ROCTAVIAN, use an effective form of birth control with your female partner to prevent pregnancy and do not donate semen.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

Using contraception<\/p>\n

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

Avoid alcohol for at least 12 months after ROCTAVIAN, and then consult with your healthcare team.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

No alcohol<\/p>\n

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

Lab visits<\/p>\n

[ANDREW SPEAKING]<\/p>\n

And lab visits\u2026<\/p>\n

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

Do not donate blood, organs, tissues or cells for transplantation.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

And I was able to make all those changes.<\/p>\n

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

LIFE AFTER ROCTAVIAN GENE THERAPY<\/p>\n

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

The DIFFERENCE between ROCTAVIAN and my previous treatments was that ROCTAVIAN is a ONE-TIME infusion.<\/p>\n

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

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

[ANDREW SPEAKING]<\/p>\n

My other therapies were multiple times per week.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

With the follow-up to the ROCTAVIAN gene therapy treatment\u2026<\/p>\n

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

Monitoring becomes less frequent over time<\/p>\n

First 6 months \u2013 weekly
\nNext 6 months \u2013 every 1 to 2 weeks
\nYear 2 \u2013 every 3 months
\nAfter Year 2 \u2013 every 6 months<\/p>\n

Follow-up tests
\nLiver enzyme test
\nA blood test that measures liver enzyme levels to see if they are elevated from your baseline.<\/p>\n

Factor VIII activity test
\nA blood test that measures Factor VIII activity levels to see how well ROCTAVIAN is working.<\/p>\n

Both of these tests can be done at the same appointment. Consider visiting the same lab or location for all tests, since results could vary between labs.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I would go to my doctor pretty frequently at the beginning and then less frequently over time. For me it was most convenient to go back to my hemophilia treatment center for labs, but I know that there are other places that you can have that done.<\/p>\n

Another part of the follow-up regimen was the lab, checking those liver enzyme levels and of course those Factor VIII levels. Keeping a close eye on those levels is really important since ROCTAVIAN works in the liver.<\/p>\n

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

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.<\/p>\n

Talk to your doctor about these side effects and what you need to do to improve and maintain your liver\u2019s health.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

My doctor did tell me that if my liver enzyme levels are high, I may need to be prescribed oral steroids temporarily to bring levels down.<\/p>\n

[ON SCREEN TEXT]<\/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

Median is the middle number in a list of numbers arranged from smallest to largest.<\/p>\n

Range shows the lowest and highest numbers in a list.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I was on steroids for about 3 months. I know that steroids have their own potential adverse events, so that\u2019s certainly something to talk to your doctor about.<\/p>\n

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

Avoid alcohol for at least 12 months after ROCTAVIAN, and then consult with your healthcare team.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

After a year, my doctor said an occasional alcoholic beverage in moderation is fine. That\u2019s important here in the bourbon capital of the world.<\/p>\n

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

[ROCTAVAIN logo]<\/p>\n

The first time I realized ROCTAVIAN was working for me<\/p>\n

Individual results may vary.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

Before ROCTAVIAN if I cut myself shaving I would have to factor up because I\u2019m someone who continued to bleed after a cut. The first story I like to tell is definitely the shaving story where I cut myself shaving, ran to the fridge to grab the factor to mix it up and, have an infusion, and before I could even go get it, uh, the bleeding had stopped. And that was kind of a\u2014kind of a breakthrough moment, kind of a, you know, we\u2019re here. It\u2019s working. That was kind of the first sign that that it really was- was doing what I hoped.<\/p>\n

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

NOW, ROCTAVIAN HELPS MY BODY MAKE ITS OWN FACTOR<\/p>\n

[ANDREW SPEAKING]<\/p>\n

At about the 6-month mark, I went to the doctor. I knew it was important to get checked, I knew it was important for them to continue to monitor me.<\/p>\n

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

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.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

I don’t know specific numbers, but I do know that my factor levels continued to rise, they hit a peak and then slowly started to decline.<\/p>\n

3 years later, my doctor told me that my body is still producing Factor VIII above the severe range and I’m still off of continuous prophylaxis.<\/p>\n

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

Andrew was part of the directly enrolled population. 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.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

Following the ROCTAVIAN infusion, I infused for 3 weeks after that, but I\u2019ve not had an infusion since then. What I couldn’t create before was Factor VIII, and now my body does it.<\/p>\n

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

[ROCTAVIAN logo]<\/p>\n

3 years after ROCTAVIAN<\/p>\n

[ANDREW SPEAKING]<\/p>\n

It was hard to get used to at first, not infusing, changing the schedule. But now, I have a new normal.<\/p>\n

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

[ROCTAVIAN logo]<\/p>\n

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.*<\/p>\n

97% Infusion reduction Throughout the 3-year follow-up period, average infusions were reduced from 136 per year before ROCTAVIAN to 5 per year after ROCTAVIAN.<\/p>\n

Average Infusions Per Year<\/p>\n

Before ROCTAVIAN\u2020 (136)
\nAfter ROCTAVIAN (5)<\/p>\n

*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. Median is the middle number in a list of numbers arranged from smallest to largest.
\n\u2020Data were collected for 6 months and those results were annualized.<\/p>\n

Andrew worked with his doctor to stop prophylaxis in the weeks following his ROCTAVIAN infusion.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

Honestly, this experience was worth it. Just to kind of lessen that number of infusions and hopefully lessen that number of bleeds.<\/p>\n

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

[ROCTAVIAN logo]<\/p>\n

IMAGINE WHAT’S POSSIBLE<\/p>\n

For 6 months after ROCTAVIAN use an effective form of birth control with your female partner to prevent pregnancy.<\/p>\n

[ANDREW SPEAKING]<\/p>\n

There’s been a big change in my life. My newborn daughter. We had our daughter two and a half years after my ROCTAVIAN gene therapy treatment. That was a part of the decision-making process on kind of waiting to start a family. My mornings now are a little bit less routine, little more time. But it’s getting up, look at the new real estate listings for the day. Drink a cup of coffee. Give all the dogs a good lap around the block. And then back to get my niece and nephew ready for school and head to work.<\/p>\n

And I would also encourage everybody to kind of do the research and talk to their doctors about ROCTAVIAN, see if it\u2019s something that\u2019s right for you.<\/p>\n

But really the only way to know your eligibility is to get that blood test done.<\/p>\n

Definitely one of the big reasons that I wanted to be a part of the clinical trial was to help other people in the community.<\/p>\n

[ONSCREEN TEXT & ANDREW SPEAKING]<\/p>\n

This is MY ROCTAVIAN EXPERIENCE.<\/p>\n

Are YOU ready to start YOURS?<\/p>\n

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

Our heartfelt thanks go out to Andrew and the other clinical trial participants, researchers, and healthcare providers who helped make this possible.<\/p>\n

What will your ROCTAVIAN story be?<\/p>\n

Talk to your healthcare team today to see if you\u2019re eligible for one-time ROCTAVIAN gene therapy.<\/p>\n

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

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

What is ROCTAVIAN?<\/strong>
\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

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