Andrew W. Transcript

[ON SCREEN TEXT]

ROCTAVIAN logo

[NARRATOR AND ON SCREEN TEXT]
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.

[ON SCREEN TEXT]
My ROCTAVIAN Experience
Meet Andrew
ROCTAVIAN clinical study participant

[ANDREW W.]
Hi, I’m Andrew. I live with severe hemophilia A, and this is my ROCTAVIAN story.

[ON SCREEN TEXT]
IMPORTANT SAFETY INFORMATION

[NARRATOR AND ON SCREEN TEXT]
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.

[ON SCREEN TEXT]
Andrew W., received ROCTAVIAN in 2018 as part of a clinical trial

[ANDREW W.]
I’m from Louisville, Kentucky, where I live with my wife, my daughter, and my niece and nephew.

[ON SCREEN TEXT]
Why I chose ROCTAVIAN

[ANDREW W.]
I 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.

[ON SCREEN TEXT]
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.

ROCTAVIAN gene therapy starts with simple eligibility tests. Talk to your healthcare team about eligibility testing for ROCTAVIAN gene therapy.

[ANDREW W.]
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.

I never imagined a day where I wouldn’t have to actively treat with factor.

[ON SCREEN TEXT]
5 years after ROCTAVIAN, more than 70% of people who had responded to ROCTAVIAN stopped—and stayed off— continuous prophylaxis

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).

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.

Prophylaxis is defined as the ongoing use of Factor VIII or another treatment to prevent bleeds.

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.

[Infographic of 70%—80 of 112 people]

[ON SCREEN TEXT]
Safety profile of ROCTAVIAN after 5 years

The safety of ROCTAVIAN is being evaluated in a clinical trial

• 134 people enrolled*
• 5 years of data so far

Long-term safety continues to be studied, and data will be collected for years.

*Included 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).

[Infographic: Safety profile of ROCTAVIAN after 5 years]

[ON SCREEN TEXT]
In the clinical study
The most common side effects of ROCTAVIAN were: (N=134)

• Nausea 32.1% of people
• Fatigue 15.7% of people
• Infusion-related reactions 9% of people
• Headache 7.5% of people
• Vomiting 6% of people
• Abdominal pain 6% of people
• Elevated liver enzyme levels that were temporary, asymptomatic, and low grade 82.8% of people experienced ALT levels above the upper limit of normal

ALT, alanine aminotransferase.

[Infographic: Side effects of ROCTAVIAN]

[NARRATOR AND ON SCREEN TEXT]
Depending on your risk factors, an improvement in Factor VIII levels may mean an increased possibility of unwanted blood clots (so called “thrombosis,” 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.

[ON SCREEN TEXT]
ROCTAVIAN INFUSION DAY

[ANDREW W.]
I remember everything about my infusion day, everything that happened that day.

[ANDREW W. AND ON SCREEN TEXT]
It took a few hours. They monitored me throughout and continued to check on me afterwards.

[ON SCREEN TEXT]
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.

[NARRATOR AND ON SCREEN TEXT]
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, 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.

[ANDREW W.]
My doctor explained to me the lifestyle changes that I would have to make…

[ON SCREEN TEXT]
For 6 months after ROCTAVIAN, use an effective form of birth control with your female partner to prevent pregnancy and do not donate semen.

[ON SCREEN TEXT]
Avoid alcohol for at least 12 months after ROCTAVIAN and then consult with your healthcare team.

****

[ANDREW W.]
Using contraception

No alcohol

And lab visits…

[ON SCREEN TEXT]
Do not donate blood, organs, tissues or cells for transplantation.

[ANDREW W.]
And I was able to make all those changes.

[ON SCREEN TEXT]
LIFE AFTER ROCTAVIAN GENE THERAPY

[ANDREW W.]
The follow-up to the ROCTAVIAN gene therapy treatment…

[ON SCREEN TEXT]
Monitoring becomes less frequent over time First 6 months—WEEKLY

Next 6 months—EVERY 1 TO 2 WEEKS Year 2—EVERY 3 MONTHS

After year 2—EVERY 6 MONTHS

[ANDREW W.]
I would go to my doctor pretty frequently at the beginning and then less frequently over time.

[ON SCREEN TEXT]
Follow up

Liver enzyme test
A blood test that measures liver enzyme levels to see if they are elevated from your baseline.

Factor VIII activity test
A blood test that measures Factor VIII activity levels to see how well ROCTAVIAN is working.

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.

[ANDREW W.]
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.

[ON SCREEN TEXT]
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.

[ANDREW W.]
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.

[ON SCREEN TEXT]
Most patients in the clinical trial required corticosteroids and the median duration of corticosteroid use was 35 weeks.

[ANDREW W.]
I was on steroids for about 3 months. I know that steroids have their own potential adverse events, so that’s certainly something to talk to your doctor about.

[ON SCREEN TEXT]
THE FIRST TIME I REALIZED ROCTAVIAN WAS WORKING FOR ME

Individual results may vary.

[ANDREW W.]
Before ROCTAVIAN if I cut myself shaving, I would have to factor up because I’m 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, the bleeding had stopped.

And that was kind of a—kind of a breakthrough moment, kind of a, you know, we’re here. It’s working. That was kind of the first sign that—that it really was—was doing what I hoped.

[ON SCREEN TEXT]
NOW, ROCTAVIAN HELPS MY BODY MAKE ITS OWN FACTOR

Individual results may vary. Not all patients respond to ROCTAVIAN.

[ON SCREEN TEXT]
At Year 5, the average Factor VIII level was 29.8, ranging from 1.5 to 321.2 in the 85 participants in the rollover population based on available data in the Phase 3 study.

[ANDREW W.]
My doctor told me that my body is still producing Factor VIII above the severe range and I’m still off of continuous prophylaxis.

[ON SCREEN TEXT]
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.

[ANDREW W.]
Following the ROCTAVIAN infusion I infused for 3 weeks after that, but I have not had an infusion since then. What I couldn’t create before was Factor VIII and now my body does it.

[ON SCREEN TEXT]
5 years after ROCTAVIAN

[Graphic: 5 years after ROCTAVIAN] [ON SCREEN TEXT]

95% infusion reduction through 5 years (n=112)

Before ROCTAVIAN
Patients averaged 136 infusions per year*^†

After ROCTAVIAN
Infusions declined to 7 per year

*The follow-up period began 5 weeks after administration and consists of a median follow- up of 5 years with a range of 1.7 to 5.2 years.

^†Baseline data were collected for 6 months and those results were annualized

[ANDREW W.]
It was hard to get used to at first, not infusing, changing the schedule. But now, I have a new normal.

[Graphic: Results are based on 112 people whose data were collected for at least 6 months before receiving ROCTAVIAN]

[ON SCREEN TEXT]
Andrew worked with his doctor to stop prophylaxis in the weeks following his ROCTAVIAN infusion.

[ANDREW W.]
Honestly, this experience was worth it, just to kind of lessen that number of infusions and hopefully lessen that number of bleeds.

[ON SCREEN TEXT AND GRAPHIC]

One infusion.
Years of freedom from prophylaxis is possible.
Individual results may vary. Not all patients respond to ROCTAVIAN.

[ON SCREEN TEXT]
For 6 months after ROCTAVIAN, use an effective form of birth control with your female partner to prevent pregnancy.

[ANDREW W.]
There’s been a big change in my life, a newborn daughter. We had our daughter 2 and a half years after my ROCTAVIAN gene therapy treatment.

[ANDREW W.]
That was a part of the decision-making process on kind of waiting to start a family.

And I would also encourage everybody to kind of do the research and talk to their doctors about ROCTAVIAN, see if it’s something that’s right for you.

[ON SCREEN TEXT AND ANDREW W.]
This is my ROCTAVIAN experience.

Are YOU ready to start yours?

[ON SCREEN TEXT]
Our heartfelt thanks go out to Andrew and the other clinical trial participants, researchers, and healthcare providers who helped make this possible.

What will your ROCTAVIAN story be?
Talk to your healthcare team today to see if you’re eligible for one-time ROCTAVIAN gene therapy.

[ROCTAVIAN logo]

[ON SCREEN TEXT AND 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:

• 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)

What is the most important information I should know about ROCTAVIAN?

ROCTAVIAN may cause serious side effects during the infusion and afterward:

• 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, light- headedness (near-fainting), fever, chills, or Talk to your doctor about what to do if you experience any side effects after you leave the infusion
• Before and regularly following administration of ROCTAVIAN, your doctor will perform blood tests to check your liver health. Make sure you obtain these blood tests during the specified time your doctor instructs you to. 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
• Patients with active Factor VIII inhibitors should not take ROCTAVIAN. Following administration your doctor will monitor you for inhibitors and you will have regular factor level testing. Talk to your doctor if you start bleeding following ROCTAVIAN, in order for your doctor to assess the need for additional tests or treatments
• 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
• 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

What should I tell my doctor before I get ROCTAVIAN?
Talk to your doctor about the following:

• Your medical conditions including:
  • Any general risk factors for unwanted blood clots and for cardiovascular disease
  • If your immune system’s ability to fight infections is reduced
  • If you have inhibitors or a history of inhibitors to Factor VIII
• All medicines you take or new medicines you plan to take, including prescription and nonprescription drugs, vitamins, herbal supplements, and vaccines
• If you have a female partner that plans to become pregnant within 6 months of treatment

What should I avoid after taking ROCTAVIAN?

• Avoid alcohol use for the first year. Talk to your doctor about how much alcohol may be acceptable after the first year
• You and any female partner must prevent becoming pregnant for 6 months. Discuss with your doctor which methods of contraception are suitable
• Do not donate semen for at least 6 months after treatment
• Do not donate blood, organs, tissues, or cells

What are the possible side effects of ROCTAVIAN?

• The most common side effects of ROCTAVIAN are:
  • Nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain
  • Changes to laboratory results from blood tests that measure your liver health and other ways your body is responding to ROCTAVIAN

What other information should I know before getting ROCTAVIAN?

• Receiving gene therapy again in the future: ROCTAVIAN is a one-time treatment
• Currently, treatment with ROCTAVIAN means you cannot receive another gene therapy for hemophilia
• Hemophilia treatment registry: 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
• Understanding the risks and benefits of ROCTAVIAN: While the majority of patients experience a benefit from ROCTAVIAN, the treatment response and duration 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. After administration, 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 any surgeries, procedures, injuries, or bleeds

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.

[ON SCREEN TEXT]
ROCTAVIAN® is a registered trademark of BioMarin Pharmaceutical Inc. ©2025 BioMarin

Pharmaceutical Inc. All rights reserved. COM-RC-0123 11/25

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