[ON SCREEN TEXT]
ROCTAVIAN logo
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.
[DAVE SPEAKING]
My name is Dave, and this is my severe hemophilia A story.
[ON SCREEN TEXT]
Meet Dave
My ROCTAVIAN experience
Received ROCTAVIAN in 2024
[DAVE SPEAKING]
I’m 39 years old.
Growing up with severe hemophilia A was challenging. I would say that there was never really an easy time.
[ON SCREEN TEXT]
Dave
[DAVE SPEAKING]
I didn’t participate in any sports—official sports that the school sponsored—just because it was too difficult to heal my injuries with the medicine that was available at the time.
[ON SCREEN TEXT]
ROCTAVIAN is indicated for adults only. ROCTAVIAN is not intended for administration in women.
[DAVE SPEAKING]
As an adult, I’m doing more traveling. I have more activities. I’m taking care of other people. So I have to make sure I really focus on taking care of myself so I’m able to do those things.
[ON SCREEN TEXT]
ROCTAVIAN is indicated for adults only.
[DAVE SPEAKING]
I have two kids, Ava and Anthony…
[ON SCREEN TEXT]
Jenna
[DAVE SPEAKING]
…and a wife, Jenna.
[JENNA SPEAKING]
I’m Jenna, and I’m Dave’s wife. We have been married for 2 years, and we met in high school.
[DAVE SPEAKING]
In 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.
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.
[JENNA SPEAKING]
I had to help Dave remember to treat some days.
[ON SCREEN TEXT]
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), or are allergic to mannitol (an inactive ingredient in ROCTAVIAN).
Please see additional safety information throughout and at the end of this video.
[DAVE SPEAKING]
So 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.
[ON SCREEN TEXT]
ROCTAVIAN did not work for everyone. Some patients did not respond to treatment or lost response to treatment.
[DAVE SPEAKING]
The steps that I took to learn more about ROCTAVIAN were just talking to my doctors.
[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.
[DAVE SPEAKING]
My doctor discussed with me the risks and benefits as well as the lifestyle changes that come with ROCTAVIAN.
[ON SCREEN TEXT]
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
[DAVE SPEAKING]
My biggest concern about taking ROCTAVIAN is that if it didn’t give me the results that I was looking for, that I would have to go back to being on prophylaxis.
[ON SCREEN TEXT]
Dave’s ROCTAVIAN story started with simple eligibility tests. Connect with your healthcare team to schedule yours today.
[DAVE SPEAKING]
The eligibility process for me was luckily very easy.
And then I made sure that my liver was healthy and that I didn’t contain any of the antibodies that ROCTAVIAN worked with to make sure that my body wouldn’t fight it when I received it.
[ON SCREEN TEXT]
Dave 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.
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.
Infusion Day
[DAVE SPEAKING]
I would describe my infusion day by just saying, “Exciting!” It was a very early morning because it’s 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.
[ON SCREEN TEXT & NARRATOR]
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.
[ON SCREEN TEXT]
Follow-ups are important to check your liver health and 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 check your liver health and measure Factor VIII levels
Follow -ups will become less frequent over time and help your healthcare team know when you can stop routine prophylaxis
[DAVE SPEAKING]
Follow-up post-infusion, I had weekly blood tests to make sure that all the levels of my liver were looking good and healthy.
[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.
[DAVE SPEAKING]
So having the weekly blood test and knowing what my factor levels were was very exciting 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. Talk to your doctor about these side effects and what you need to do to improve and maintain your liver’s health. Your follow-up schedule may need to change if you are taking corticosteroids.
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.
[DAVE SPEAKING]
And that went on for about 6 months, and then I went to every other week for the following 6 months.
I did take steroids to help my liver kind of control the changes that were going on.
[ON SCREEN TEXT]
Life after ROCTAVIAN
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.
[DAVE SPEAKING]
ROCTAVIAN has impacted my treatment routine because I don’t have a routine anymore.
[ON SCREEN TEXT]
Dave 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.
[DAVE SPEAKING]
After my ROCTAVIAN infusion, I was on prophylaxis for a month until my doctor told me I didn’t have to do prophylaxis anymore.
[ON SCREEN TEXT]
5 years after ROCTAVIAN, more than 70% of people (80 out of 112) had responded then stopped—and stayed off—continuous prophylaxis*†
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’s right for you.
*Data were collected for 6 months, and those results were annualized.
†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.
[DAVE SPEAKING]
After ROCTAVIAN, my factor levels began to rise and peaked after a year.
Everybody has different results.
[ON SCREEN TEXT]
Nausea 32.1% of people
Fatigue 15.7% of people
Headache 7.5% of people
Infusion-related reactions 9% of people
Vomiting 6% of people
Abdominal pain 6% of people
Elevated liver enzyme levels that were temporary and asymptomatic
82.8% of people experienced ALT levels above the upper limit of normal
ALT=alanine aminotransferase.
[NARRATOR SPEAKING]
In the clinical study, the most common side effects were nausea, fatigue, headache, infusion-related reactions, vomiting, abdominal pain, and elevated liver enzyme levels.
[ON SCREEN TEXT]
Results 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.
Before ROCTAVIAN*†
Patients averaged 136 infusions per year
95% Infusion reduction through 5 years (n=112)
After ROCTAVIAN*
Infusions declined to an average of 7 per year
Throughout the 5-year follow-up period, average infusions were reduced from 136 per year before ROCTAVIAN to 7 per year after ROCTAVIAN.
*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.
†Data were collected for 6 months, and those results were annualized.
[DAVE SPEAKING]
For somebody who’s considering ROCTAVIAN, I would say do your research. Make sure you have all the information.
But for me, I would say go for it.
[ON SCREEN TEXT]
While 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.
[JENNA SPEAKING]
It is quite a process, but it was nice to see, and just be supportive along the way no matter what the results are.
[DAVE SPEAKING]
So 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.
[ON SCREEN TEXT]
Spontaneity can be your focus
One infusion. Years of freedom from continuous prophylaxis is possible.
[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.
[BioMarin logo]
ROCTAVIAN® is a registered trademark of BioMarin Pharmaceutical Inc. © 2025 BioMarin Pharmaceutical Inc.
All rights reserved. COM-RC-0079 11/25
Contraindications: Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol.
Infusion-related reactions including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis.
Hepatotoxicity: The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient’s ability to receive corticosteroids and/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy.
Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor Factor VIII activity levels since ALT elevation may be accompanied by a decrease in Factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis.
It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease Factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and/or Factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions.
Thromboembolic Events: Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated Factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their Factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event.
Factor VIII Inhibitors and Monitoring for Inhibitors. The safety and effectiveness of ROCTAVIAN in patients with prior or active Factor VIII inhibitors have not been established. Patients with active Factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for Factor VIII inhibitors (neutralizing antibodies to Factor VIII). Test for Factor VIII inhibitors especially if bleeding is not controlled, or plasma Factor VIII activity levels decrease.
Monitor Factor VIII using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived Factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous Factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with Factor VIII activity levels ≤5 IU/dL and evidence of bleeding, taking into account the stability of Factor VIII levels since the previous measurement.
Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for Factor VIII activity assay interference during the transition period.
Malignancy: The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (eg, hepatitis B or C, nonalcoholic fatty liver disease, chronic alcohol consumption, nonalcoholic steatohepatitis, advanced age) with regular liver ultrasound (eg, annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100.
Most Common Adverse Reactions: Most common adverse reactions (incidence ≥5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain. Most common laboratory abnormalities (incidence ≥10%) were ALT, AST, LDH, CPK, Factor VIII activity levels, GGT, and bilirubin >ULN. Patients also experienced adverse reactions from corticosteroid use.
Isotretinoin, Efavirenz, and HIV-Positive Patients. Isotretinoin is not recommended in patients who are benefiting from ROCTAVIAN. Efavirenz is not recommended in patients treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection.
Females and Males of Reproductive Potential. ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women or regarding lactation. For 6 months after administration of ROCTAVIAN, men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and men must not donate semen.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100.
Please see additional safety information in the Prescribing Information.
Indication
ROCTAVIAN® (valoctocogene roxaparvovec-rvox) is indicated for the treatment of adults with severe hemophilia A (congenital Factor VIII deficiency with Factor VIII activity <1 IU/dL) without antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.