Mepsevii

Indicated For

Mucopolysaccharidosis VII (MPS VII, Sly syndrome)

About This Medication

# UltraCare Patient Assistance Program: How to Get Mepsevii at Low or No Cost ## About This Program The UltraCare Patient Assistance Program, offered by Ultragenyx Pharmaceutical Inc., helps patients access Mepsevii (vestronidase alfa-vjbk) when cost is a barrier to treatment. Mepsevii is an enzyme replacement therapy used to treat Mucopolysaccharidosis VII (MPS VII, also called Sly syndrome), a rare genetic disorder affecting the body's ability to break down certain substances in cells. ## What is Mepsevii and Who Needs It? Mepsevii is a recombinant human lysosomal beta-glucuronidase enzyme indicated for pediatric and adult patients with MPS VII. The medication is administered as an intravenous infusion every two weeks at a dose of 4 mg/kg of body weight under medical supervision. Patients must be at least 5 months of age to receive treatment. MPS VII is caused by mutations in the gene encoding the beta-glucuronidase enzyme. Without adequate enzyme function, harmful substances accumulate in cells, leading to progressive symptoms affecting mobility, organ function, and quality of life. Mepsevii works by replacing the missing or deficient enzyme. ## Who Qualifies for UltraCare? ### Insurance Status Requirements You may qualify for the UltraCare Patient Assistance Program if you are: - **Uninsured**: You have no active health insurance coverage - **Underinsured**: You have health insurance but face significant out-of-pocket costs for Mepsevii, including high copayments, coinsurance, or deductibles ### Income Eligibility While specific income thresholds for the UltraCare program are not publicly detailed, patient assistance programs typically consider household income relative to the federal poverty level. Income limits vary by family size. Contact the program directly for current income eligibility guidelines specific to your household situation. ### Other Eligibility Requirements To qualify, you must: - Have a documented diagnosis of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) - Be prescribed Mepsevii by or in consultation with a physician specializing in metabolic disease, lysosomal storage disorders, genetics, or related fields - Not have contraindications to Mepsevii therapy - Be a U.S. resident ## How to Apply ### Step 1: Gather Required Documentation Before applying, collect the following documents: - Proof of diagnosis (medical records confirming MPS VII diagnosis) - Current prescription for Mepsevii from your healthcare provider - Proof of income (recent tax returns, pay stubs, or benefit statements) - Proof of insurance status (insurance card, denial letter, or proof of uninsured status) - Identification (driver's license or state ID) - Proof of U.S. residency ### Step 2: Submit Your Application The UltraCare program accepts applications through multiple methods. Contact Ultragenyx Pharmaceutical Inc. directly to determine the most convenient application method for your situation: - Phone: Contact the program for the current phone number - Online: Visit the Ultragenyx website for online application options - Mail: Request a paper application form - Healthcare Provider: Your doctor's office may submit on your behalf ### Step 3: Application Review Once submitted, your application will be reviewed by the program team. They will verify your eligibility based on insurance status, income, diagnosis, and other criteria. ### Step 4: Receive Your Decision You will be notified of the program's decision. If approved, you will receive information about how to access your medication at reduced or no cost. ## Timeline and Medication Delivery Processing times for applications vary. Contact the UltraCare program directly for current processing timeframes. Once approved, Mepsevii will be delivered according to the program's delivery methods, typically coordinated with your healthcare provider's infusion center or pharmacy. ## Important Considerations ### Medical Supervision Required Mepsevii must be administered under the supervision of a healthcare professional with the capability to manage anaphylaxis. Infusions occur every two weeks and require: - Premedication with a non-sedating antihistamine 30-60 minutes before infusion - Careful monitoring during the 4-hour infusion period - Observation for at least 60 minutes after infusion completion ### Anaphylaxis Risk Anaphylaxis is a serious risk with Mepsevii, potentially occurring as early as the first dose. Appropriate medical support must be available during administration. Inform your healthcare provider immediately of any signs of allergic reaction. ## What If Your Application Is Denied? If your application is denied, you have several options: 1. **Request reconsideration**: Ask the program to review your application if circumstances have changed 2. **Explore other assistance**: Investigate other patient assistance programs, nonprofit organizations, or disease-specific foundations that support MPS VII patients 3. **Contact your healthcare provider**: Your doctor may have resources or alternative funding options 4. **Verify insurance coverage**: If you have insurance, work with your insurer to understand coverage options and appeal processes ## Reauthorization and Ongoing Support If you are approved for assistance, you may need to reauthorize your participation periodically. The program will notify you of any reauthorization requirements and provide instructions for renewal. ## Disclaimer This guide provides general information about the UltraCare Patient Assistance Program. Program details, eligibility criteria, and benefits may change. For the most current and accurate information, contact Ultragenyx Pharmaceutical Inc. directly or visit their official website. This information is not a substitute for professional medical or financial advice. Consult your healthcare provider and the program directly regarding your specific situation.

Program information last verified: March 30, 2026