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, an enzyme normally required for the breakdown of certain complex carbohydrates known as as glycosaminoglycans (GAGs).
“The Making of NAGLAZYME” (galsulfase) highlights the treatment’s journey from discovery to approval and includes powerful perspectives from MPS VI patients, BioMarin employees, researchers and practitioners.
- First specific approved therapy for the treatment of MPS VI
- Approved in multiple countries
- Designated an orphan drug in the United States and internationally
- Manufactured and commercialized by BioMarin
In May 2005, Naglazyme received marketing approval from the U.S. Food and drug Administration for the treatment of MPS VI patients. Shortly thereafter, in January 2006, Naglazyme received marketing authorization in the European Union. Naglazyme was designated an orphan drug in the United States with seven years market exclusivity, and an orphan drug in the European Union with 10 years exclusivity. Naglazyme has also been approved in several countries in Europe and Asia, as well as Australia.
BioMarin is commercializing Naglazyme worldwide.
NAGLAZYME (galsulfase) is indicated for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome). NAGLAZYME has been shown to improve walking and stair- climbing capacity.
Important Safety Information
Severe and life-threatening allergic reactions can occur during NAGLAZYME (galsulfase) infusions and up to 24 hours after infusion. Typical signs of an allergic reaction include shock, difficulty breathing, wheezing, swelling of the throat, and low blood pressure. If a severe allergic reaction occurs during infusion, the infusion should be stopped immediately and you should receive medical attention. Contact your doctor or get medical help right away if you develop any severe symptoms after infusion.
In clinical trials, most patients developed antibodies to NAGLAZYME treatment. There was no clear relationship between antibody formation and the safety or effectiveness of NAGLAZYME.
Serious and severe infusion reactions are associated with NAGLAZYME, including hives, chest pain, rash, abdominal pain, difficulty breathing, swelling, fever, and eye irritation. You should receive medication such as antihistamines before NAGLAZYME infusions to reduce the risk of infusion reactions. If an infusion reaction occurs, the infusion should be slowed or stopped and you may be given additional medication.
The most common side effects of NAGLAZYME seen in clinical trials were rash, pain, hives, fever, itching, chills, headache, nausea, vomiting, abdominal pain and difficulty breathing. The most common side effects requiring medical attention are infusion-related effects.
These are not all of the possible side effects with NAGLAZYME. Talk to your doctor if you have any symptoms that bother you or that do not go away.
NAGLAZYME is a prescription medicine. Before treatment with NAGLAZYME, it is important to discuss your medical history with your doctor. Tell your doctor if you are taking any medication and if you are allergic to any medicines. Your doctor will decide if NAGLAZYME is right for you. If you have questions or would like more information about NAGLAZYME, contact your doctor.
Spinal cord damage may occur due to the natural MPS VI disease process. Signs of spinal cord injury include back pain, loss of bladder and bowel control, numbness, and paralysis. Contact your doctor immediately if you develop any of these symptoms.
To report SUSPECTED ADVERSE REACTIONS contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.
Please see accompanying full Prescribing Information or visit www.Naglazyme.com.