Naglazyme® (galsulfase) for MPS VI
Naglazyme® (galsulfase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI), an inherited life-threatening lysosomal storage disorder caused by a deficiency of the lysosomal enzyme
N-acetylgalactosamine 4-sulfatase. Naglazyme provides a recombinant version of this enzyme to individuals diagnosed with MPS VI.
Naglazyme at a Glance
- Approved in the United States and European Union for the treatment of MPS VI
- Designated an orphan drug in the United States and European Union
- Manufactured and commercialized by BioMarin
Regulatory Status
Naglazyme is approved in both the United States and the European Union for the treatment of MPS VI. The U.S. Food and Drug Administration (FDA) approved the therapy on May 31, 2005 and subsequently, the European Commission approved Naglazyme on January 24, 2006. As the first drug approved for MPS VI, the FDA and EC have both designated Naglazyme as an orphan drug, conferring seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union.
Commercialization Plan
BioMarin is commercializing Naglazyme in the United States and European Union.
Important Safety Information
The most common adverse events in patients treated with Naglazyme were headache, fever, joint pain, vomiting, upper respiratory infections, abdominal pain, diarrhea, ear pain, cough, and ear infections. Severe reactions included swollen blood vessels, low blood pressure, difficulty breathing, respiratory distress, stopping breathing, and hives. The most common symptoms of infusion reactions included fever, chills/shakes, headache, rash, and mild to moderate hives. Nausea, vomiting, elevated blood pressure, chest pain, abdominal pain, malaise, and joint pain were also reported. No patients discontinued Naglazyme treatment because of reactions. Nearly all patients developed antibodies as a result of the treatment, but the level of immune response did not correlate with the severity of the adverse reaction. Because antihistamine use may increase the risk of stopping breathing, airways should be checked to ensure they are not blocked or obstructed. Treatment may be delayed if you have a fever or respiratory illness.
Please see patient product information. If you have any questions about this information, please talk with your doctor.
