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Products

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, European Union, and at least 7 other countries in the rest of the world for the treatment of MPS VI
  • Designated an orphan drug in the United States and internationally
  • Manufactured and commercialized by BioMarin

Regulatory Status

In May 2005, Naglazyme received marketing approval from the US Food and drug Administration for the treatment of MPS VI patients. Shortly thereafter, in January 2006, Naglazyme received approval in the European Union. Naglazyme was designated an orphan drug in the US and EU, which grants market exclusivity for 7 and 10 years, respectively. Naglazyme has also been approved in Australia, Brazil, Croatia, Japan, South Korea, Switzerland, and Belarus. Naglazyme has been granted orphan drug designation in Australia, Japan, South Korea and Switzerland as well.

Commercialization Plan

BioMarin is commercializing Naglazyme worldwide.



 

Naglazyme is indicated for patients with mucopolysaccharidosis VI (MPS VI). Naglazyme has been shown to improve walking and stair-climbing capacity.

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.

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Naglazyme is available by prescription only. To learn more, please visit www.naglazyme.com for full prescribing information. If you have any questions about this information, please talk with your doctor.