Benefits of NAGLAZYME® (galsulfase)

In MPS VI, the buildup of GAGs can lead to entire organs or bodily systems being harmed. Normalizing GAG levels may help to slow the decline associated with this condition. NAGLAZYME® (galsulfase) provides the missing enzyme in MPS VI so that cells can break down and reduce the buildup of GAGs.

If left untreated, individuals with MPS VI can develop severe complications that can lead to physical difficulties, including:

  • Reduced endurance or walking distance
  • Difficulty breathing

Endurance is an important measure in people with MPS VI because it can show how well your heart, lung, and musculoskeletal systems are working together. MPS VI experts recommend using timed walking and stair-climbing tests to assess endurance.

Measuring endurance and GAG levels in the urine (uGAGs) can help doctors keep track of changes over time and see how well NAGLAZYME is working.

Quick improvement with NAGLAZYME

In the 6-month primary study, individuals taking NAGLAZYME on a weekly basis significantly improved their endurance and reduced uGAG levels.

Improved endurance

Individuals taking NAGLAZYME experienced a 23% improvement in walking distancea and a 38% improvement in stair-climbing.

aResults were taken from the 6-minute time point of the 12-minute walk test.

Reduced uGAGs

Regardless of their state of MPS VI progression, all patients being treated with NAGLAZYME showed a 75% reduction in uGAG levels.
 

 

Weekly infusions lead to long-lasting improvement

According to a 10-year follow up study, we now know the benefits of NAGLAZYME can be maintained over the long-term and are consistent with the results of the primary study. This study examined endurance, lung function, and uGAG levels in patients who had been taking NAGLAZYME for up to 10 years (the average time on NAGLAZYME therapy was 6.8 years).

Improved endurance

Results from the long-term follow-up study demonstrated that individuals who were on NAGLAZYME long-term continued to experience a 21% increase in walking distance.b

bResults from the 6-minute walk test.

Helped lungs work better

Continued treatment with NAGLAZYME has been shown to improve lung function in people with MPS VI, which can make breathing easier.
 

Reduced uGAGs

Long-term adherence to NAGLAZYME treatment showed sustained reduction in uGAG levels that were 87.9% lower than at baseline.
 

 

If you would like to learn more about NAGLAZYME clinical studies, talk to your doctor or contact BioMarin RareConnections™.

About_MPS_VI_video
play-btn
play-btn_hov
View video: Dr. Whitely discusses NAGLAZYME.
Next Page

Important Safety Information

Indication

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 full Prescribing Information.