Brineura

Indicated For

neuronal ceroid lipofuscinosis type 2 (CLN2 disease), TPP1 deficiency, Batten disease

About This Medication

# BioMarin RareConnections Patient Guide: How to Get Brineura (cerliponase alfa) at Low or No Cost Brineura (cerliponase alfa) is a specialized enzyme replacement therapy for pediatric patients with CLN2 disease, and BioMarin RareConnections offers co-pay assistance and support to make it more affordable for eligible families.[1][2] This guide explains eligibility, application steps, and resources to access treatment smoothly. ## About Brineura and CLN2 Disease **Brineura (cerliponase alfa)** is the only FDA-approved therapy to slow the loss of ability to walk or crawl (ambulation) in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency—a rare, progressive form of Batten disease.[1][2][7] Approved in 2017, it works by delivering the missing TPP1 enzyme directly into the brain's fluid via intraventricular infusion every two weeks, addressing the underlying cause of the disease.[2] CLN2 disease typically appears in children ages 2-4, causing seizures, vision loss, motor decline, and cognitive issues. Early treatment with Brineura can help preserve motor function, but it requires a surgically implanted intraventricular access device (ICVD) and administration at specialized centers by experienced neurologists.[2][7][8] It's not suitable for patients with active ICVD complications like infections, leaks, or brain shunts.[2] Brineura is infused over 4+ hours biweekly at 300 mg (or age-appropriate dose), with close monitoring for anaphylaxis or hydrocephalus risks—always review full Prescribing Information.[1][2] ## Who Qualifies for Assistance? BioMarin RareConnections provides **one-on-one support** including insurance navigation, co-pay help, and financial options for eligible patients.[1][3] The primary program is the **Brineura Co-Pay Assistance Program**, targeting **commercially insured patients** (private insurance, not government plans like Medicare/Medicaid).[1][5] - **Key eligibility**: U.S. or Puerto Rico residents with commercial insurance prescribed Brineura for CLN2 disease.[5] - Eligible patients may pay **as little as $0** for co-pays after enrollment.[1][5] - A separate **BioMarin Patient Assist Program** offers free medication for those who cannot afford it, based on financial need (details via RxAssist).[4] - Government-insured patients (Medicare, Medicaid) may get prior authorization support but not co-pay aid from this program.[5][9] **Note**: Specific income thresholds are not publicly detailed; case managers assess eligibility individually.[1][4] ## Income Eligibility Breakdown Detailed FPL-based thresholds aren't specified online, but assistance prioritizes financial hardship.[4] Co-pay help is not needs-based but requires commercial insurance.[5] Contact BioMarin for personalized review. | Household Size | Estimated Threshold | Notes | |---------------|-------------------|-------| | Individual | Case-by-case | Commercial insurance primary; financial aid for uninsured/underinsured.[1][4] | | Couple | Case-by-case | Free med program for low-income.[4] | | Family of 3 | Case-by-case | Co-pay as low as $0 if eligible.[1] | | Family of 4+ | Case-by-case | Puerto Rico included.[5] | ## Insurance Requirements - **Commercial insurance required** for co-pay program; helps cover out-of-pocket costs set by your plan.[1][5] - **Medicare/Medicaid**: Coverage possible with prior authorization (PA), but no co-pay assistance—use patient assist for free meds if eligible.[6][7][9] - Insurers often require CLN2 diagnosis confirmation, baseline motor scores, and specialist oversight.[7][8] - BioMarin helps with PA, appeals, and reimbursement.[1][8] ## Step-by-Step Application Process Enrollment is simple and doctor-involved:[1][5] 1. **Discuss with your doctor**: Confirm CLN2 diagnosis, ICVD placement, and Brineura suitability. Get baseline assessments (e.g., CLN2 Impairment Score).[7][8] 2. **Complete Patient Consent Form (PCF)**: Authorizes sharing info with BioMarin and your insurer.[1] 3. **Doctor submits Patient Enrollment Form (PEF)**: Online or via phone; includes prescription and medical details.[1][5] 4. **BioMarin Case Manager contacts you**: Reviews coverage, explains options (call 866-906-6100, Mon-Fri 8AM-8PM ET).[1][5] 5. **Enroll in Co-Pay Program**: If eligible, get flyer/terms; activation not needed.[1][5] 6. **Hospital setup**: Plan ICVD surgery and infusion at a specialized center (takes time).[2][8] Download forms from BioMarin RareConnections site.[1] ## Timeline and Delivery - **Enrollment to contact**: Days after submission.[1] - **PA approval**: 30-45 days standard; expedited for urgent cases.[8] - **Initial therapy**: Up to 6 months; reauth every 6-12 months with progress docs.[6][7] - **Delivery**: Via specialty pharmacy to infusion center; case manager coordinates.[1][3] - **Infusions**: Every 2 weeks ongoing.[2] ## Alternatives if Denied or Ineligible - **Appeal denial**: BioMarin aids appeals (50% overturn rate in some states); resubmit with more evidence like motor scores.[8] - **Patient Assist Program**: Free Brineura for uninsured/low-income.[4] - **State aid**: Medicaid PA possible with diagnosis proof.[6] - **Other support**: GoodRx for co-pay cards; specialty centers for grants.[9] - No biosimilars available.[7] ## Important Disclaimer This guide is for informational purposes based on public sources as of 2026; eligibility varies. Always consult your doctor, BioMarin (866-906-6100), or www.biomarin-rareconnections.com. Terms apply; program may change. Not medical/financial advice—review PI for risks like anaphylaxis.[1][2]

Program information last verified: March 30, 2026