Vestronidase Alfa — Membership Pricing

Other medications in Metabolic

• Asfotase Alfa
• Cerliponase Alfa
• Eliglustat
• Inotersen
• Miglustat
• Nitisinone
• Patisiran
• Sapropterin

About Vestronidase Alfa

Vestronidase alfa, known commercially as VIMIZIM®, offers a vital enzyme replacement therapy for individuals living with Maroteaux-Lamy Syndrome (MPS IVA). This life-changing medication, while essential, often comes with significant costs. TheDrugClub is dedicated to helping our members explore options to manage these expenses and access the care they need, making treatment more attainable.

Vestronidase alfa is a specialized medication primarily used to treat Maroteaux-Lamy Syndrome (MPS IVA), a rare, inherited lysosomal storage disorder. Individuals with MPS IVA lack a crucial enzyme called N-acetylgalactosamine-6-sulfatase. Without this enzyme, complex sugar molecules called glycosaminoglycans (GAGs), specifically keratan sulfate and chondroitin sulfate, cannot be properly broken down and accumulate in various tissues and organs throughout the body. This accumulation leads to a wide range of debilitating symptoms, including skeletal abnormalities, joint stiffness, respiratory problems, heart valve disease, and reduced endurance.

How Vestronidase Alfa Works

Vestronidase alfa functions as an enzyme replacement therapy (ERT). It is a recombinant form of the human N-acetylgalactosamine-6-sulfatase enzyme, manufactured to mimic the body's natural enzyme. When administered intravenously, vestronidase alfa helps to replace the missing enzyme in patients with MPS IVA. By providing this functional enzyme, the medication aids in the breakdown of accumulating GAGs, reducing their storage in cells. This action can help to alleviate symptoms, improve walking capacity, and potentially slow the progression of the disease, offering a significant improvement in the quality of life for affected individuals.

Typical Costs Associated with Vestronidase Alfa

As a highly specialized, biologic medication for a rare disease, vestronidase alfa is one of the most expensive treatments available. The typical annual cost can range from several hundred thousand dollars to over a million dollars, depending on the patient's weight, dosage, and treatment frequency. This extraordinary cost makes it largely inaccessible for most individuals without substantial financial assistance or comprehensive insurance coverage. Factors like ongoing administration fees and associated medical care can further add to

The information above is for educational purposes only and does not constitute medical advice. Consult your healthcare provider before making medication decisions.

What Vestronidase Alfa is approved for (FDA label)

MEPSEVII is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Limitations of Use The effect of MEPSEVII on the central nervous system manifestations of MPS VII has not been determined. MEPSEVII is a recombinant human lysosomal beta glucuronidase indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Limitations of Use The effect of MEPSEVII on the central nervous system manifestations of MPS VII has not been determined. ( 1 )

Source: Read the full FDA label on DailyMed. Label effective date: November 18, 2025.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Boxed warning for Vestronidase Alfa (FDA label)

WARNING: ANAPHYLAXIS Anaphylaxis has occurred with MEPSEVII administration, as early as the first dose [see Warnings and Precautions ( 5.1 )] , therefore appropriate medical support should be readily available when MEPSEVII is administered. Closely observe patients during and for 60 minutes after MEPSEVII infusion [see Dosage and Administration ( 2.2 ), Warnings and Precautions ( 5.1 )]. Immediately discontinue the MEPSEVII infusion if the patient experiences anaphylaxis [see Dosage and Administration ( 2.2 ), Warnings and Precautions ( 5.1 )]. WARNING: ANAPHYLAXIS See full prescribing information for complete boxed warning. Anaphylaxis has occurred with MEPSEVII administration, as early as the first dose ( 5.1 ), therefore appropriate medical support should be readily available when MEPSEVII is administered. Closely observe patients during and for 60 minutes after MEPSEVII infusion ( 2.2 , 5.1 ). Immediately discontinue the MEPSEVII infusion if the patient experiences anaphylaxis ( 2.2 , 5.1 ).

Source: FDA prescribing information (DailyMed). Set ID: 925d4e21-fd98-474d-a34d-a0b3558ed750. SPL document ID: 6e20f052-5a5f-41cd-ab55-97b8a9bf6562. Label effective date: November 18, 2025. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Dosage forms and strengths for Vestronidase Alfa (FDA label)

Injection: 10 mg/5 mL (2 mg/mL) as a colorless to slightly yellow liquid in a single-dose vial. Injection: 10 mg/5 mL (2 mg/mL) in a single-dose vial ( 3 )

Source: FDA prescribing information (DailyMed). Set ID: 925d4e21-fd98-474d-a34d-a0b3558ed750. SPL document ID: 6e20f052-5a5f-41cd-ab55-97b8a9bf6562. Label effective date: November 18, 2025. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Adverse reactions for Vestronidase Alfa (FDA label)

The following serious adverse reactions are described below and elsewhere in the labeling: Anaphylaxis [see Warnings and Precautions ( 5.1 )] Most common adverse reactions (≥1 patient) are: Infusion site extravasation, diarrhea, rash, anaphylaxis, infusion site swelling, peripheral swelling and pruritus. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ultragenyx at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Source: FDA prescribing information (DailyMed). Set ID: 925d4e21-fd98-474d-a34d-a0b3558ed750. SPL document ID: 6e20f052-5a5f-41cd-ab55-97b8a9bf6562. Label effective date: November 18, 2025. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Contraindications for Vestronidase Alfa (FDA label)

None. None ( 4 )

Source: FDA prescribing information (DailyMed). Set ID: 925d4e21-fd98-474d-a34d-a0b3558ed750. SPL document ID: 6e20f052-5a5f-41cd-ab55-97b8a9bf6562. Label effective date: November 18, 2025. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Warnings and precautions for Vestronidase Alfa (FDA label)

5.1 Anaphylaxis Anaphylaxis to MEPSEVII was reported in 2 of 20 patients in the clinical program [see Adverse Reactions ( 6.1 )] . These reactions occurred during MEPSEVII infusion and were observed as early as the first dose of MEPSEVII for one patient. Manifestations included respiratory distress, cyanosis, decreased oxygen saturation, and hypotension. The two patients with anaphylaxis to MEPSEVII during the clinical trials had one occurrence each and tolerated subsequent infusions of MEPSEVII, without recurrence. Anaphylaxis can be life-threatening. MEPSEVII should be administered under the supervision of a healthcare professional with the capability to manage anaphylaxis. Patients should be observed for 60 minutes after MEPSEVII administration. If severe systemic reactions occur, including anaphylaxis, immediately discontinue the MEPSEVII infusion and provide appropriate medical treatment. Prior to discharge, inform patients of the signs and symptoms of anaphylaxis and instruct them to seek immediate medical care if symptoms occur.

Source: FDA prescribing information (DailyMed). Set ID: 925d4e21-fd98-474d-a34d-a0b3558ed750. SPL document ID: 6e20f052-5a5f-41cd-ab55-97b8a9bf6562. Label effective date: November 18, 2025. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.