Have questions about ROCTAVIAN that aren’t answered here?
Have additional questions about ROCTAVIAN?
Connect with our team of experts for more information.
Have questions about ROCTAVIAN that aren’t answered here?
Andrew S., dosed with ROCTAVIAN in
2018 as part of a clinical trial.
ROCTAVIAN is a one-time infusion that was studied in the longest and largest Phase 3 gene therapy clinical trial for severe hemophilia A—now with 5 years of data demonstrating:
Important first steps to treatment with ROCTAVIAN are:
ROCTAVIAN is administered at hemophilia treatment centers (HTCs) throughout the United States. These centers are not only familiar with the logistics of the infusion and follow-up monitoring, but also the coverage process.
You can help patients connect with a hemophilia treatment center using our HTC Finder Tool.
We may be able to help! While there are a number of HTCs prescribing ROCTAVIAN throughout the United States, travel-related logistics may be a concern. Enroll your patients in BioMarin RareConnections™ to allow our team of experts to provide support and expedite access to ROCTAVIAN.
ROCTAVIAN is approved for adults with severe hemophilia A who do not have antibodies to the AAV5 virus, which is determined by a blood test. Learn more about how to test for eligibility for ROCTAVIAN.1
AAV5 DetectCDx™ is a trademark of ARUP Laboratories.
The decision to make a change in treatment can be an emotional one, but our team of experts at BioMarin RareConnections™ is here to provide support to you and your patients every step of the way. Once patients enroll, they can get questions about ROCTAVIAN answered, receive one-to-one educational and product support, and learn about the experiences of other patients who chose ROCTAVIAN.
The one-time cost of ROCTAVIAN is covered by most insurance plans. If you have questions about costs or coverage, we can help. Our team of experts at BioMarin RareConnections™ is here to provide support and expedite access to ROCTAVIAN.
If you have questions about coverage or billing concerns for ROCTAVIAN, we can help. Enroll your patients in BioMarin RareConnections™ to allow our team of experts to provide support and expedite access to ROCTAVIAN.
BioMarin began clinical trials of ROCTAVIAN in 2015,* making it the most extensively studied hemophilia A gene therapy. An ongoing, prospective, Phase 3, open-label, single-dose, single-arm, multinational study began in 2017 (N=134), and data continue to be collected.1,3,4 Review the 5-year data.
*The first patient received ROCTAVIAN in a Phase 1/2 clinical trial.3
ROCTAVIAN has been proven to deliver Factor VIII activity levels for 5 years for most patients. In the clinical trial, some patients had increased Factor VIII levels, but it was determined that they had not responded or lost response to ROCTAVIAN.
The most common adverse reactions (≥5%) to ROCTAVIAN were nausea, fatigue, headache, vomiting, abdominal pain, and infusion-related reactions.1
The safety profile of ROCTAVIAN was evaluated in 134 patients in the Phase 3 study (112 rollover and 22 directly enrolled). Long-term safety continues to be evaluated in the long term follow-up study. Learn more about the safety of ROCTAVIAN.
Administration of ROCTAVIAN does not eliminate the ability to return to prophylaxis. In the clinical trial, some patients had increased Factor VIII levels, but it was determined that they had not responded or lost response to ROCTAVIAN. These patients returned to prophylaxis.1
Take a closer look at the clinical trial data for ROCTAVIAN.
If you have questions or concerns about data, coverage, and billing for ROCTAVIAN, or want to connect a patient with an HTC, we can help. Enroll your patients in BioMarin RareConnections™ to allow our team of experts to provide support and expedite access to ROCTAVIAN.
Hear firsthand experiences from people who chose to move forward with single-infusion ROCTAVIAN.
References:
1. ROCTAVIAN® (valoctocogene roxaparvovec-rvox). Prescribing information. BioMarin International, Ltd.; 2023. 2. Klamroth R, Hayes G, Andreeva T, et al. Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with hemophilia A. Hum Gene Ther. 2022;33(7-8):432-441. doi:10.1089/ hum.2021.287
Connect with our team of experts for more information.
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.