Roctavian

Roctavian Patient Guide: How to Get Gene Therapy Treatment Support

About This Program

Roctavian (valoctocogene roxaparvovec-rvox) is an innovative gene therapy developed by BioMarin Pharmaceutical Inc. for patients with severe hemophilia B. BioMarin offers patient support programs to help eligible patients access this treatment and manage associated costs.

About Roctavian

Roctavian is a one-time gene therapy treatment designed for adults with severe hemophilia B who do not have inhibitors to Factor IX. This groundbreaking treatment works by delivering a functional Factor IX gene to help your body produce the clotting factor it needs. Unlike traditional hemophilia treatments that require regular infusions, Roctavian aims to provide long-term improvement in clotting ability.

Who Can Get Help

BioMarin's patient support programs are designed to assist patients with:

• Commercial Insurance: Patients with active commercial prescription insurance coverage
• Financial Barriers: Those facing high out-of-pocket costs, copayments, or coinsurance
• Verified Medical Need: Patients with a valid prescription from their healthcare provider for an FDA-approved indication

Patients without commercial insurance or those on government-funded programs (Medicare, Medicaid, VA, Tricare) may have different eligibility requirements and should contact BioMarin directly.

Income Eligibility

BioMarin's patient assistance programs do not publish specific income thresholds. Eligibility is determined on a case-by-case basis considering:

• Your household income and financial situation
• Insurance coverage and out-of-pocket costs
• Medical necessity as documented by your healthcare provider
• State-specific regulations

To learn if you qualify based on your specific financial circumstances, you'll need to complete the enrollment process with BioMarin RareConnections.

Insurance Requirements

Commercial Insurance: Most BioMarin assistance programs require active commercial prescription insurance coverage. The program helps reduce your out-of-pocket expenses (copayments, coinsurance, and deductibles).

Government Programs: If you receive coverage through Medicare, Medicaid, VA, Tricare, or other government-funded programs, you are generally not eligible for BioMarin's co-pay assistance programs. However, you may qualify for other support services.

State Restrictions: Patients in certain states (Massachusetts and Rhode Island) have limitations on drug administration co-pay support. Patients in Michigan, Minnesota, and Rhode Island have restrictions on laboratory services co-pay support.

Step-by-Step Application Process

Step 1: Contact BioMarin RareConnections

Reach out to BioMarin's patient support team to begin the enrollment process:

• Phone: 1-866-906-6100
• Email: support@biomarin-rareconnections.com
• Hours: Monday–Friday, 7 AM–4 PM (PT)

A case manager will be assigned to help guide you through the entire process, from insurance verification to ordering your medication.

Step 2: Complete Enrollment Forms

You'll need to submit:

• BioMarin RareConnections Patient Registration Form (if not previously completed)
• Proof of commercial insurance
• Financial information for eligibility determination
• Your healthcare provider's prescription and Statement of Medical Necessity

Step 3: Preliminary Screening and Approval

BioMarin will review your information and determine preliminary eligibility. If approved, you'll receive:

• A conditional approval letter
• A copay ID number
• Instructions for next steps, including how to submit claims

Step 4: Treatment and Claim Submission

Once you receive Roctavian treatment:

• Your healthcare provider submits a claim to your insurance and to BioMarin
• BioMarin processes the claim and makes a final eligibility determination
• Upon approval, payment is issued to cover your out-of-pocket costs

Step 5: Ongoing Support

Your BioMarin case manager continues to support you with:

• Prior authorization coordination with your insurance
• Specialty pharmacy coordination for medication delivery
• Refill assistance and reauthorization
• Answers to questions about your treatment

Timeline and Delivery

Processing Time: The exact timeline from application to approval is not specified by BioMarin. However, the preliminary screening process typically takes several business days to a few weeks, depending on the completeness of your application and insurance verification.

Delivery Method: Roctavian is administered as an intravenous infusion at a specialized treatment center or hospital. BioMarin coordinates with specialty pharmacies and healthcare providers to ensure proper delivery and administration of your treatment.

Financial Assistance Timeline: Once your claim is submitted and processed, BioMarin issues payment to cover approved out-of-pocket expenses. The timing depends on insurance processing and claim submission procedures.

What Happens If You're Denied

If your application is denied:

1. Ask Why: Contact BioMarin RareConnections to understand the specific reason for denial
2. Review Your Information: Verify that all submitted information is accurate and complete
3. Reapply: If circumstances have changed or additional information is available, you may reapply
4. Explore Alternatives: Ask about other BioMarin support programs or state pharmaceutical assistance programs
5. Appeal: Ask if there is an appeal process available

Your case manager can help you understand your options and next steps.

Reauthorization

BioMarin requires annual reauthorization of eligibility. This means:

• You must confirm your continued eligibility each year
• Your financial situation and insurance coverage will be reviewed
• You'll need to provide updated information as requested
• Failure to reauthorize may result in loss of program benefits

BioMarin will contact you when reauthorization is needed.

Important Restrictions and Limitations

• One-Time Therapy: Roctavian is a one-time gene therapy treatment, so ongoing refills are not applicable in the traditional sense
• State Limitations: Certain states have restrictions on what types of costs can be covered
• Insurance Changes: If you transition to government-funded insurance, you must notify BioMarin immediately and will no longer be eligible for the co-pay assistance program
• Program Changes: BioMarin reserves the right to modify or terminate the program at any time
• Annual Limits: Assistance is subject to annual maximums and program terms

Getting More Information

BioMarin RareConnections

• Phone: 1-866-906-6100
• Email: support@biomarin-rareconnections.com
• Website: biomarin-rareconnections.com

Your healthcare provider and specialty pharmacy can also provide information about BioMarin's support programs.

Disclaimer

This guide provides general information about BioMarin's patient support programs as of March 2026. Program eligibility, benefits, and requirements are subject to change. Income thresholds, specific coverage amounts, and detailed eligibility criteria are not publicly specified and must be determined through direct contact with BioMarin RareConnections. This information is not a guarantee of program enrollment or financial assistance. Always verify current program details directly with BioMarin before making treatment decisions. Consult with your healthcare provider about whether Roctavian is appropriate for your medical condition.