Andrew S. 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.

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MY ROCTAVIAN EXPERIENCE
MEET ANDREW
ROCTAVIAN clinical study participant

[ANDREW S.]
My name is Andrew. I live in Fort Collins, Colorado. I was born with severe hemophilia A.

My brother’s 20 months older than me and also has severe hemophilia A. It was great to have a partner, have an ally in hemophilia. And 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 am happily married almost 15 years.

I have three kids, a boy and two little girls. I just cherish them and love being a dad. We spend a lot of time outdoors—biking and playing soccer and just generally exploring.

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?

[ON SCREEN TEXT]
Andrew received his ROCTAVIAN infusion in October 2018.

[ANDREW S.]
I first heard about ROCTAVIAN from my brother.

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CONSIDERING ROCTAVIAN

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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 S.]
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.

[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 70% of people]

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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).

[Graphic: Safety profile of ROCTAVIAN after 5 years]

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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.

[ANDREW S.]
To learn more about ROCTAVIAN, I talked to my doctor, I talked to my nurse, my family. 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.

[ON SCREEN TEXT AND 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

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Andrew’s ROCTAVIAN story started with simple eligibility tests
Connect with your healthcare team to schedule yours today

[ANDREW S.]
I was so excited when I learned I was eligible to receive ROCTAVIAN. We got the tests back, and I was so excited to move forward.

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Things to consider before treatment

• Your healthcare team will ask you to not drink alcohol for the first 12 months after treatment
• Check with them before taking any medicines, herbal products, or supplements
• Use an effective form of birth control with your female partner for 6 months after ROCTAVIAN
• Do not donate blood, organs, tissues, or cells for transplantation

[ANDREW S.]
There were lifestyle considerations I had to make for a year. 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.

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, 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.

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LIFE AFTER ROCTAVIAN

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Follow-ups are important to help you see when your body starts to make its own Factor VIII

• For the first 6 months after your infusion, you’ll need weekly blood tests to measure your Factor VIII levels
• Follow-ups will become less frequent over time and help your healthcare team know when you can stop routine prophylaxis

[ANDREW S.]
Monitoring for my ROCTAVIAN gene therapy involved weekly visits to the hemophilia treatment center. And I would go to the treatment center and get an update on my factor level. We’d also get an update on where my liver enzymes were at, because they carefully monitor your liver health.

[ON SCREEN TEXT AND NARRATOR]
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.

[ANDREW S.]
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 get the inside scoop on what ROCTAVIAN gene therapy was doing for me.

[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.

Most patients in the clinic trial required corticosteroids and the median duration of corticosteroid use was 35 weeks.

[ANDREW S.]
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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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.

[ON SCREEN TEXT]
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 S.]
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 gotten a shot three times a week. And that first week with no shot, I would bump myself and I wouldn’t bleed.

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

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

Before ROCTAVIAN
Patients averaged 136 infusions per year*^†

After ROCTAVIAN
Infusions declined an average of 7 per year

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

[ANDREW S.]
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 something. I can just go.

This is my experience. Everyone’s experience may be different.

[ON SCREEN TEXT]
PROPHYLAXIS-FREE FOR YEARS

Patients who did not respond or lost response to ROCTAVIAN treatment can return to prophylaxis.

[Graphic: PROPHYLAXIS-FREE FOR YEARS]

[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 (n=85), the average Factor VIII level was 29.8%, with a range of 1.5 to 321.2 at Year 5.

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 S.]
Advice I would give to somebody 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 S.]
Think of where you could go, if you’re not thinking about infusions.

[ON SCREEN TEXT]
One infusion.
Years of freedom from continuous prophylaxis is possible.

[ON SCREEN TEXT]
Individual results may vary.
Not all patients respond to ROCTAVIAN.

[ANDREW S.]
It’s pretty cool to know that my body is making its own factor now, with ROCTAVIAN gene therapy.

[ROCTAVIAN logo]

[ON SCREEN TEXT AND NARRATOR]

Indication and Important Safety Information What is ROCTAVIAN?

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 shivering. 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.

ROCTAVIAN® is a registered trademark of BioMarin Pharmaceutical Inc. ©2025 BioMarin Pharmaceutical Inc.

All rights reserved. COM-RC-0124 11/25

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