Naglazyme (galsulfase) (Galsulfase SOLUTION) 5 mg/5mL

Naglazyme (galsulfase) is an FDA-approved orphan drug manufactured by BioMarin Pharmaceutical Inc. for the treatment of Mucopolysaccharidosis VI (MPS VI), a rare lysosomal storage disorder. It was designated as an orphan drug due to its indication in this ultra-rare genetic condition affecting fewer than 200,000 individuals in the United States.

Strength

5 mg/5mL

Pack Size

1 VIAL in 1 CARTON (68135-020-01) / 5 mL in 1 VIAL

Manufacturer / TM Owner

BioMarin Pharmaceutical Inc.

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Indications & Clinical Uses

Naglazyme is indicated for patients with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome), a rare autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme arylsulfatase B (ASB). This enzyme deficiency leads to accumulation of glycosaminoglycans (GAGs) in multiple tissues, causing progressive skeletal deformities, cardiac valve disease, respiratory compromise, and reduced life expectancy. Naglazyme is a recombinant form of human arylsulfatase B that works by replacing the deficient enzyme, thereby reducing GAG accumulation. The drug is administered intravenously and is used to slow disease progression and improve clinical outcomes in pediatric and adult patients with MPS VI. Treatment typically begins early in the disease course to maximize therapeutic benefit and prevent irreversible organ damage.

Side Effects

Common adverse effects of Naglazyme include infusion-related reactions such as fever, chills, headache, nausea, and vomiting, which may occur during or shortly after administration. Serious adverse events include anaphylaxis, angioedema, and severe hypersensitivity reactions. Patients may develop antibodies to galsulfase, potentially reducing drug efficacy. Other reported effects include tachycardia, dyspnea, and rash. Premedication with antihistamines and corticosteroids is often recommended to mitigate infusion reactions. Regular monitoring of vital signs during infusion, assessment of clinical response, and periodic evaluation of antibody formation are essential components of therapy management.

Precautions & Warnings

Naglazyme is contraindicated in patients with known hypersensitivity to galsulfase or any excipient. Caution is warranted in patients with compromised cardiac or respiratory function due to underlying MPS VI pathology. Severe infusion reactions may occur and require immediate medical intervention; emergency equipment should be available during administration. Use in pregnancy has not been adequately studied; risk-benefit assessment is necessary. Pediatric patients require careful dosing and monitoring. Patients with renal or hepatic impairment should be evaluated individually, though specific dose adjustments are not established. Immunocompromised patients may have altered responses to therapy.

Avoid Interactions

No major drug-drug interactions have been formally documented with Naglazyme. However, concurrent use with other medications affecting immune function or infusion reactions should be carefully monitored. Antihistamines and corticosteroids used for premedication do not significantly interact with galsulfase. Patients receiving other enzyme replacement therapies or substrate reduction therapies for lysosomal storage disorders should have coordinated medical oversight. Healthcare providers should review all concurrent medications and supplements with patients prior to Naglazyme initiation.

Frequently Asked Questions

Naglazyme is used to treat Mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy Syndrome), a rare genetic lysosomal storage disorder. It replaces the deficient enzyme arylsulfatase B, helping to reduce the accumulation of harmful substances in the body and slow disease progression. Treatment aims to improve clinical outcomes and quality of life in affected patients.

Naglazyme is administered as an intravenous infusion, typically once weekly. The infusion is given over approximately 4 hours in a healthcare setting where medical personnel can monitor for adverse reactions. Patients are usually premedicated with antihistamines and corticosteroids to reduce the risk of infusion-related reactions.

Common side effects include infusion-related reactions such as fever, chills, headache, nausea, and vomiting. Some patients may experience tachycardia, dyspnea, or rash. Serious but less common reactions include anaphylaxis and angioedema, which require immediate medical attention.

Naglazyme is manufactured by BioMarin Pharmaceutical Inc., a biopharmaceutical company specializing in rare genetic diseases and lysosomal storage disorders.

Yes, Naglazyme received FDA orphan drug designation due to its indication in Mucopolysaccharidosis VI, a rare disease affecting fewer than 200,000 individuals in the United States. This designation provides regulatory incentives and support for development of treatments for rare, life-threatening conditions.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated: 17 Jun 2026

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