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

On average, 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, patients treated with NAGLAZYME showed an average 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™.

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View video: Dr. Whitely discusses NAGLAZYME.
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INDICATION AND IMPORTANT SAFETY INFORMATION

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.

What is the most important information I should know about NAGLAZYME?

Severe and life-threatening allergic reactions, including anaphylaxis, can occur during NAGLAZYME infusions and up to 24 hours after infusion. These reactions can occur in people receiving NAGLAZYME for the first time or in people who have previously received NAGLAZYME without having an allergic reaction.

Your doctor will tell you about the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and when to seek immediate medical care. Signs of anaphylaxis can include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. If a severe allergic reaction (e.g., anaphylaxis) 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.

What are the most common side effects of NAGLAZYME?

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.

What else should I know about NAGLAZYME?

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.

Call your doctor for medical advice about side effects. You may report side effects to BioMarin at 1-866-906-6100 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

Please see full accompanying Prescribing Information, with important warning for risk of anaphylaxis or visit www.Naglazyme.com.

INDICATION AND IMPORTANT SAFETY INFORMATION

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.

What is the most important information I should know about NAGLAZYME?

Severe and life-threatening allergic reactions, including anaphylaxis, can occur during NAGLAZYME infusions and up to 24 hours after infusion. These reactions can occur in people receiving NAGLAZYME for the first time or in people who have previously received NAGLAZYME without having an allergic reaction.

Your doctor will tell you about the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and when to seek immediate medical care. Signs of anaphylaxis can include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. If a severe allergic reaction (e.g., anaphylaxis) 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.

What are the most common side effects of NAGLAZYME?

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.

What else should I know about NAGLAZYME?

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.

Call your doctor for medical advice about side effects. You may report side effects to BioMarin at 1-866-906-6100 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

Please see full accompanying Prescribing Information, with important warning for risk of anaphylaxis or visit www.Naglazyme.com.