Naglazyme

Indicated For

MPS VI, Mucopolysaccharidosis VI

About This Medication

# BioMarin RareConnections Patient Guide: How to Get Naglazyme at Low or No Cost Naglazyme (galsulfase) is an enzyme replacement therapy for patients with **Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome)**, a rare genetic disorder that affects the body's ability to break down certain substances, leading to buildup that can cause skeletal abnormalities, joint stiffness, heart and lung problems, and reduced endurance[1][6][7]. **BioMarin RareConnections** is the patient assistance program from **BioMarin** that helps eligible patients access Naglazyme through support with billing, insurance navigation, infusion center coordination, and financial aid like co-pay assistance[1][2][4]. ## About Naglazyme and MPS VI MPS VI is a lysosomal storage disorder where the enzyme **N-acetylgalactosamine 4-sulfatase** is deficient, causing **glycosaminoglycans (GAGs)** to accumulate in tissues and organs[6][7]. This leads to symptoms like short stature, coarse facial features, hearing loss, corneal clouding, and progressive mobility issues. Naglazyme provides the missing enzyme intravenously once weekly, improving **walking and stair-climbing capacity**, endurance, pulmonary function, and reducing urinary GAG levels with consistent use[5][6][7]. **Important Safety Note**: Naglazyme carries a **black box warning** for **hypersensitivity reactions, including anaphylaxis**, which can occur during or up to 24 hours after infusion. Start treatment in a healthcare setting with monitoring and resuscitation equipment available. Common side effects include rash, pain, hives, headache, fever, and itching[3][5][7]. Always discuss risks with your doctor. The recommended dose is **1 mg/kg body weight weekly**, infused over **at least 4 hours**. It can be given at an **infusion center, doctor's office, hospital, pharmacy, or even at home** after coordination[1][3][8]. Home infusions involve receiving vials from a specialty pharmacy, storing them refrigerated, and having a nurse administer[3]. ## Who Qualifies for BioMarin RareConnections? This program supports patients with a valid Naglazyme prescription facing access barriers. It offers **personalized case management** for coverage, financial needs, education, and infusion logistics[2][4]. Key eligibility focuses on **commercially insured patients** needing **co-pay assistance**; specific income guidelines are **not publicly disclosed**[program details]. It's **not for government-funded insurance** like Medicare or Medicaid[program details]. **Income Eligibility Breakdown** Since exact Federal Poverty Level (FPL) thresholds are not disclosed, eligibility is determined case-by-case. Contact the program for personalized assessment. | Household Size | Income Threshold | FPL % | Notes | |---------------|------------------|-------|-------| | Individual | Not disclosed | N/A | Case-by-case review; proof of income required[program details] | | Couple | Not disclosed | N/A | Co-pay help for commercial insurance[program details] | | Family of 3 | Not disclosed | N/A | Financial need evaluated individually | | Family of 4 | Not disclosed | N/A | Guidelines not public | ## Insurance Requirements You must have **commercial prescription insurance** (private plans, not government programs like Medicare, Medicaid, VA, or Tricare)[program details][2][4]. The program helps with **prior authorizations, appeals, and billing** to streamline coverage[1][2]. Uninsured or underinsured patients may qualify for additional support—call to confirm[4]. ## Step-by-Step Application Process 1. **Get a Prescription**: Discuss Naglazyme with your doctor if you have MPS VI. They confirm diagnosis and prescribe[1][6]. 2. **Contact BioMarin RareConnections**: Call **(866) 906-6100** to start enrollment. A case manager guides you[1][3][program details]. 3. **Complete Forms**: Sign the **Patient Consent Form (PCF)** online or via phone. Your doctor submits the **Patient Enrollment Form (PEF)**[2][4][10]. 4. **Submit Documents**: Provide **proof of income**, **insurance information**, **prescription**, and **doctor-completed application**[program details]. 5. **Approval and Coordination**: Case manager helps find an **infusion center** (or home setup), handles insurance, and ensures delivery[1][3]. 6. **Start Treatment**: Attend first infusion in a monitored setting. Weekly infusions follow[5][8]. Applications are handled **by phone**; no online portal listed[program details]. **Reauthorization is required** periodically to continue benefits[program details][5]. ## Timeline and Delivery Processing time varies; expect case manager contact soon after calling. Infusion setup depends on insurance approval—could take days to weeks[1][2]. Naglazyme arrives at your **hospital, doctor's office, pharmacy, or home via specialty pharmacy**[3][program details]. For home: Refrigerate vials (discard if damaged or unrefrigerated >24 hours), apply numbing cream if prescribed, stay hydrated[3]. Infusion takes ~4 hours; monitor for reactions[8]. ## If Denied or Alternatives If denied, your case manager assists with **appeals** or alternative funding options[2][4]. No **biosimilar alternatives** exist for Naglazyme[program details]. Explore independent foundations, state programs, or clinical trials via your doctor. Lifelong weekly therapy is key for benefits—discuss interruptions with your physician[5]. ## Disclaimer This guide is for informational purposes based on available program details as of general knowledge. Eligibility, benefits, and processes can change; **always verify with BioMarin RareConnections at (866) 906-6100** or your healthcare provider. Not medical advice—consult professionals for treatment decisions. BioMarin does not guarantee coverage or assistance[1][2][program details]. (Word count: 1028)

Program information last verified: March 30, 2026