For U.S. Healthcare ProfessionalsFor U.S. Patients & Caregivers
Select a treatment for support

We’re here to help.
For assistance, please call
1-833-ROCTAVIAN (1-833-762-8284)
Monday-Friday, 8AM-8PM ET

For assistance, please call 1-833-ROCTAVIAN (1-833-762-8284)

Enroll your ROCTAVIAN patients to access support for their one-time infusion

ROCTAVIAN is the only FDA-approved gene therapy for eligible adults with severe hemophilia A. This one-time infusion is matched by personalized product and education support for patients and centers from BioMarin RareConnections™.

Our dedicated team of Case Managers and Therapy Access Managers provides patients with ROCTAVIAN eligibility testing and help with product access support and your center with reimbursement education. Learn more about our support services.

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.

Administration of ROCTAVIAN is contraindicated in

  • 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
  • patients with known hypersensitivity to mannitol
Enrolling your patients in BioMarin RareConnections early allows our team to provide support from the start, which could help expedite access to ROCTAVIAN.

If you are ready to prescribe, complete the ROCTAVIAN Patient Enrollment Form (PEF) via QUICK ENROLL.

The PEF serves as the prescription for ROCTAVIAN.
Computer iconQUICK ENROLL e-enrollment
or
Download iconDownload the PEF
To explore support options for eligible commercially insured patients*, such as laboratory testing support and co-pay assistance, download and complete the Laboratory and Financial Support Form (LFSF) and have your patient complete the Patient Consent Form (PCF).
Download iconDownload the LFSF
+
Download iconDownload the PCF
or
Email iconEmail PCF to your patient
ROCTAVIAN logo
Full Prescribing Information
ROCTAVIAN.com/hcp
We’re here to help. For assistance, please call 1-833-ROCTAVIAN (1-833-762-8284) Monday-Friday, 8AM-8PM ET
*ROCTAVIAN Patient Support Programs are covered only for those patients with commercial prescription insurance coverage for products that meet eligibility criteria. Offer not valid for prescriptions, administration, or related labs reimbursed, in whole or in part, by any federal, state, or government-funded insurance programs (for example, Medicare, Medicare Advantage, Medigap, Medicaid, VA, DoD, or TRICARE), for cash-paying patients, where product, administration, or related lab are not covered by patient’s commercial insurance, where patient’s commercial insurance plan reimburses them for the entire cost of their prescription drug, administration, and/or related labs, or where prohibited by law or by the patient’s health insurance provider. Patients who are residents of certain states (MA or RI) are not eligible for drug administration co-pay support. Patients who are residents of certain states (MI, MN, or RI) are not eligible for laboratory services co-pay support or travel-related expenses. If at any time a patient begins receiving prescription drug, administration, or related lab coverage under any such federal, state, or government-funded healthcare program, patient will no longer be able to use ROCTAVIAN Patient Support Programs and patient must notify BioMarin RareConnections™ at 1-833-ROCTAVIAN (1-833-762-8284) to stop participation. Patients may not seek reimbursement for the value of the out-of-pocket expense amount covered by the ROCTAVIAN Patient Support Programs from any third-party payer, whether public or private. ROCTAVIAN Patient Support Programs are valid ONLY for qualifying patients residing in the 50 U.S. states or in Puerto Rico with commercial insurance who have a valid prescription for an FDA-approved indication for the qualifying BioMarin therapy. ROCTAVIAN Patient Support Programs are not health insurance. Offer may not be combined with any other rebate, coupon, or offer. Co-payment assistance under the ROCTAVIAN Patient Support Programs is not transferable. BioMarin Pharmaceutical Inc. reserves the right to rescind, revoke, or amend the ROCTAVIAN Patient Support Programs without notice. Patient/caregiver certifies responsibility for complying with applicable limitations, if any, of any commercial insurance and reporting receipt of program rewards, if necessary, to any commercial insurer. ROCTAVIAN Patient Support Programs are subject to termination or modification at any time. Some restrictions apply.
BioMarin RareConnections provides financial navigation to help patients gain support for laboratory testing and access to ROCTAVIAN. Learn more about our services and explore the following resources:
VOXZOGO Coverage Authorization Guide
A comprehensive resource including sample Letter of Medical Necessity for accessing VOXZOGO treatment for your patients.
Download the guide
VOXZOGO Sample Letter of Medical Necessity
This sample letter can be used as a reference when drafting your letter of medical necessity.
Download the letter
VOXZOGO Patient Support Program for Clinics Flyer
A guide to available BioMarin Patient Support Program services.
Download the flyer
ROCTAVIAN Reimbursement Guide​
A comprehensive resource including a sample Letter of Medical Necessity for accessing ROCTAVIAN for your patients.
Download the guide
ROCTAVIAN Laboratory and Financial Support Form​

Complete and submit this form to BioMarin RareConnections to explore laboratory support and financial options for your patients

Download the form
ROCTAVIAN Patient Support Program

A brochure explaining our comprehensive Patient Support Program to assist patients

Download the brochure
Email to your patient

COMING SOON

BioMarin RareConnections and Specialty Pharmacy Patient Pathway for Clinics Flyer
An outline of the HUB and specialty pharmacy patient pathway.
Download the flyer
BioMarin RareConnections and Specialty Pharmacy Patient Pathway for Caregivers Flyer
A patient-friendly pathway you can download and provide to your patient’s family.
Download the flyer
VOXZOGO Co-Pay Assistance Flyer
A flyer describing the VOXZOGO Co-pay Assistance Program for eligible patients.
Download the flyer
Email to your patient
Specialty Pharmacy / Distributor Contact Sheet for HTCs​

An at-a-glance reference sheet for contacting specialty pharmacies/distributors

Download the sheet

COMING SOON

rspf-icon
ROCTAVIAN Laboratory Support Program​

A flyer explaining pre-infusion eligibility and post-infusion follow-up testing support for qualifying patients

Download the flyer
Email to your patient
ROCTAVIAN Co-Pay Assistance Flyer

A flyer explaining co-pay assistance and other available support for qualifying patients

Download the flyer
Email to your patient

COMING SOON

Specialty Pharmacy Contact Sheet
An at-a-glance reference sheet for contacting specialty pharmacies.
Download the sheet
BioMarin RareConnections and Specialty Pharmacy Patient Pathway for Caregivers Flyer
A patient-friendly pathway you can download and provide to your patient’s family.
Download the flyer
VOXZOGO Co-Pay Assistance Flyer
A flyer describing the VOXZOGO Co-pay Assistance Program for eligible patients.
Download the flyer
Email to your patient

Indication and Important Safety Information

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.

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 and Important Safety Information

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.

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.