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ROCTAVIAN CLINICAL
STUDY RESULTS

Learn how doctors can confirm eligibility with simple blood tests

AM I ELIGIBLE?

Andrew W. received ROCTAVIAN in 2018 as part of a clinical study.

ROCTAVIAN is indicated for adults only.

Now with 5 years of clinical data

Studied in the longest and largest gene therapy clinical study for severe hemophilia A*

  • ROCTAVIAN is a one-time treatment that helps people make their own Factor VIII, so they may be able to†:
    • Maintain Factor VIII levels in the mild range or higher
    • Experience bleed control for years
    • Stop and stay off continuous prophylaxis for years

Now with 5 years of safety profile data

*At FDA approval, ROCTAVIAN had the longest and largest Phase 3 clinical study for gene therapy in severe hemophilia A.
†Individual Factor VIII levels produced and durability of levels reached can vary. ROCTAVIAN did not work for everyone. Some patients did not respond to treatment or lost response to treatment.

5 years after ROCTAVIAN, the average Factor VIII activity level was in the mild hemophilia range*

  • Factor VIII levels can be measured using either a one-stage assay (shown here) or a chromogenic assay. One-stage results consistently show higher Factor VIII levels
  • 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 29.8% at Year 5
  • Factor VIII activity levels are based on definitions from the World Federation of Hemophilia. Here, we define mild as 5% to less than 40% and near-normal to normal as 40% to less than or equal to 150%
  • Some people did not respond to treatment or lost response to treatment. It is not possible to predict if and how much you may benefit from ROCTAVIAN

Standard deviation (SD) shows how spread out the data are from the average.
*The follow-up period began 5 weeks or more after administration and consisted of a median follow-up of 5 years with a range of 1.7 to 5.2 years.

You could maintain Factor VIII activity levels in the mild range or higher for years*


*Here, we define Factor VIII activity levels: mild as 5% to less than 40% and near-normal to normal as 40% to less than or equal to 150%. Some patients did not respond to treatment or lost response to treatment. It is not possible to predict if and how much you may benefit from ROCTAVIAN. Some patients had elevated Factor VIII levels but didn’t respond to ROCTAVIAN.
†Factor VIII levels can be measured using either a one-stage assay (shown here) or a chromogenic assay. One-stage results consistently show higher Factor VIII levels.
‡Available data from rollover population (n=112) at week 260.
§Patients in the clinical trial experienced Factor VIII levels of <1%. Under the 5-year analysis of available, valid data, the range was 1.5 to 321.2.

ROCTAVIAN improved bleed control vs baseline for 5 years with a single infusion

  • Before ROCTAVIAN, the average (mean) annualized bleeding rate (ABR) was 5.4 (6.9). After ROCTAVIAN, the average ABR dropped to 3.9 (7.9)
  • Results are based on 112 people whose data were collected for at least 6 months while they were taking prophylaxis. These data were compared with data from the 5-year period after they received ROCTAVIAN*


*The follow-up period began 5 weeks or more after administration and consisted of a median follow-up of 5 years with a range of 1.7 to 5.2 years.
†Median is the middle number in a list of numbers arranged from smallest to largest.
‡19 of 112 people (17%) returned to continuous prophylaxis after ROCTAVIAN, with a median start time at 984 (range: 33 to 1467) days. An ABR of 35 was added to account for the periods when these people were on prophylaxis regardless of the patients’ actual bleed rate.

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It’s possible to live with severe hemophilia A that may not include continuous prophylaxis.

– Dave N., received ROCTAVIAN in 2024.
ROCTAVIAN is indicated for adults only.

WATCH DAVE’S STORY

You could be free from prophylaxis for years*

5 years after ROCTAVIAN, more than 70% of people (80 out of 112) had responded to ROCTAVIAN then 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 5-year follow-up period‡

After ROCTAVIAN, your doctor will monitor your lab results and talk to you about whether you can stop prophylaxis and whether you should start prophylaxis again if stopped. In ROCTAVIAN clinical studies, patients who did not respond to treatment or lost response to treatment were able to resume prophylaxis.

Your doctor will also discuss with you whether and how you should treat for any surgeries, procedures, injuries, or bleeds.

*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 consisted 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.
§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.

Health-related quality-of-life changes were measured over 5 years*

The Haemo-QoL-A (Haemophilia-Specific Quality of Life Questionnaire) is designed to measure health-related quality of life specifically for people with hemophilia

  • Haemo-QoL-A consists of a Total Score and 6 domain scores: Physical functioning, Role functioning, Consequences of bleeding, Worry, Emotional impact, and Treatment concern
  • Each domain measures specific aspects of HRQoL for people with hemophilia
  • Participants answer each question on a scale from 0, “none of the time,” to 5, “all of the time.” For analysis, this scale is averaged and converted to 0–100, with higher scores indicating higher HRQoL or less impairment
  • Study limitations: The ROCTAVIAN Phase 3 study was a single-arm study, which may impact the interpretation of patient-reported outcome findings. The absence of a comparator arm may result in an under- or overestimation of treatment effect. Patient-reported nature of data may impact the reliability of findings. Since formal hypothesis testing was completed after the Year 2 data cutoff per the statistical analysis plan, all data reported here are for descriptive purposes

Mean (SD) Haemo-QoL-A Total Score(n=112)

ROCTAVIAN clinical study details

The longest and largest Phase 3 gene therapy clinical study in severe hemophilia A*

Get safety information from the ROCTAVIAN clinical study.

*At FDA approval, ROCTAVIAN had the longest and largest Phase 3 clinical study for gene therapy in severe hemophilia A.

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Indication and 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)

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.

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.