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Keeping your treatment on track

Enzyme replacement therapy (ERT) is lifelong.

In order for you to increase the chances of benefiting from the treatment, NAGLAZYME® (galsulfase) must be infused regularly, on a weekly basis. You may notice some changes in how you feel right away. Other effects may take longer. Even if you don’t notice a change at first, it’s important to keep up with your treatments. If therapy stops, GAGs will again build up, and symptoms may return.1

Interesting Fact

In a study of patients receiving NAGLAZYME for 96 weeks, patients received 98% of scheduled infusions and saw sustained improvements in endurance.2,3

It’s important to keep realistic expectations about the treatment1:

  • Remember that results of NAGLAZYME treatment vary, and some improvements occur over a long period of time
  • Efficacy of ERT for MPS VI seen in the clinical studies was measured over a 6‐month period

Keep appointments with your entire care team

In addition to ERT, you will likely need symptom‐specific care from other doctors. Your cardiologist, orthopedist, ophthalmologist, or other specialist will need to see you on a regular basis to assess you for possible issues and offer treatments to ease specific symptoms.4

Keep a treatment journal

It’s a good idea to keep a calendar or diary to track your NAGLAZYME treatments. Although you may notice some changes soon, other changes may happen slowly, over weeks or months. Having a record will help you see progress clearly over time. This can help you stay motivated to keep up your treatments. Keep track of your other symptom‐specific treatments and appointments in the same journal. Here are some pointers:

  • To track your treatments, get a special calendar or diary just for this purpose
  • Mark down all your treatment appointments
  • Each day, write down your level of energy, flexibility, and range of motion
    • Note if you have an infection or don’t feel perfectly well
  • Use different colors and symbols to show different things so that you can see any trends at a glance
  • Use your journal to report any new symptoms or concerns with your geneticist and other doctors

Benefits of consistent treatment

In MPS VI, the buildup of glycosaminoglycan (GAG) causes many of the problems that patients face. NAGLAZYME provides the missing enzyme in MPS VI so that cells can break down and get rid of GAGs. It reduces and stabilizes the level of GAGs and may help to slow the decline associated with this condition.1,5

Treatment tested over the long term

Long‐term studies have been done in patients receiving NAGLAZYME. These studies examined walking, how well patients’ lungs worked, and the level of GAGs in patients who had been receiving NAGLAZYME for 6 months to 10 years (the average time on NAGLAZYME therapy was 6.8 years).1-3

In this study, the benefits of NAGLAZYME continued for patients who stayed on treatment. This included being able to walk farther, increased lung function, and a lower level of GAGs in the urine.1-3

The transition to adult care

As MPS VI patients age, it is important to plan the transition to adulthood, to ensure that they continue to get the treatment and support they currently have with their pediatric specialists and parents. This transition must be tailored to each patient’s specific needs, so that:

  • Those who can take over their own care may have the tools they need to do so
  • Those with limited capacities may have the appropriate care and services in place to support them

Planning needed

Before transition planning can begin, the doctor and parents need to review and discuss a patient’s capacities to determine what he or she will be able to handle. It is important to remember that this process is very gradual. The pediatric geneticist or other clinician coordinating the MPS VI patient’s care will oversee the transition until the patient has achieved his or her capabilities and is integrated into the adult system. The coordinating clinician will work with the parents to draw up an action plan to ease the process.

Interesting Fact

The National Health Care Transition Center offers a helpful readiness assessment tool.
Visit the center’s website to learn more.

Treatment frequently asked questions (FAQs)

What is NAGLAZYME?

NAGLAZYME is a kind of treatment called enzyme replacement therapy, or ERT. NAGLAZYME is a protein that is identical to the natural enzyme that is missing in MPS VI. NAGLAZYME reduces the GAG buildup that leads to MPS VI symptoms. In clinical studies, NAGLAZYME has been shown to help with endurance. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before. Long‐term studies in patients who had been taking NAGLAZYME for 6 months to 10 years (the average time on NAGLAZYME therapy was 6.8 years) have shown that benefits continued when patients stayed on therapy.1-3

What is NAGLAZYME used for?

NAGLAZYME is a treatment for people with MPS VI. NAGLAZYME provides the enzyme needed to break down and get rid of the excess GAGs that cause symptoms of MPS VI.1 Learn more

Can I take NAGLAZYME with other medications?

Always tell your doctor about other medications before you start a new treatment. Your doctor needs to know about any drugs you are taking now, as well as anything you might have stopped taking recently.1

Tell your doctor about all medicines, both prescription and over‐the‐counter. Also tell your doctor about vitamins or other dietary supplements you take.1

How long does a NAGLAZYME infusion take?

The infusion itself takes about 4 hours.1 Learn more 

Will it hurt?

Starting the IV can cause some pain or discomfort. Sometimes people have reactions to the medication, such as headache or nausea. If you have any problems with your infusion, talk to your doctor or nurse. They may be able to help you feel better.1 Learn more

What if I’m scared of needles?

Talk with your doctor. You might be able to get a numbing cream so the needle won’t hurt. Most people get over this fear after getting their first or second infusion. Also, focus on the fact that the needle is delivering the enzyme that you need to maintain your treatment.1  Learn more

How will I feel during infusions?

Some patients feel fine and use the time to read or do quiet activities. Other patients may feel sleepy or tired. This is often due to the premedications. If you find you are sleepy and would rather not be, talk with your doctor. You may have other premedication options.

It is possible you may have a side effect from NAGLAZYME. If you don’t feel well for any reason during an infusion, let the nurse know.1 Learn more

What are possible side effects of NAGLAZYME?

With all medications, there is a chance of side effects. This is true of NAGLAZYME too.1

Severe and life‐threatening allergic reactions, including anaphylaxis, can occur during NAGLAZYME infusions and up to 24 hours after infusion. Allergic reactions can occur in people receiving NAGLAZYME for the first time or in people who have previously received NAGLAZYME without having an allergic reaction. 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.1

Because NAGLAZYME is given by infusion, there are reactions that may happen as a result of the infusion. In clinical trials, some people experienced serious and severe infusion reactions like 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.1

If an infusion reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. For most patients, these problems went away when the infusion was stopped or slowed down. In some cases, patients were given another medicine, such as an antihistamine or pain reliever, to help.1

In clinical studies, the most common side effects of NAGLAZYME 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‐associated effects. These are not all of the possible side effects of NAGLAZYME. You should discuss with your doctor if you have any symptoms that are bothering you or do not go away.1

View additional NAGLAZYME safety information

How long will I have to take NAGLAZYME?

MPS VI is a lifelong disease, so NAGLAZYME is a lifelong treatment.1

What if I miss a treatment?

Talk with your doctor or infusion center about scheduling your next dose. Learn more

What changes can I expect from NAGLAZYME?

NAGLAZYME provides the enzyme the body needs to break down and get rid of GAGs. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before.1 Learn more

Will insurance cover me?

NAGLAZYME is an important treatment for all people with MPS VI. The experts at BioMarin RareConnections™ will help you with access to insurance coverage. All you have to do is call them at 1-866-906-6100. Learn more

Are there support programs and services to help me?

The maker of NAGLAZYME, BioMarin, has a service for people with MPS VI. It’s called BioMarin RareConnections, and it is focused on helping you get access to therapy.

This service is available at no cost and provides confidential and personalized support to assist with your healthcare needs. The goal of this service is to help you navigate the healthcare system and get access to NAGLAZYME therapy as well as other resources to help you manage MPS VI. Just call 1‐866‐906‐6100 or email support@biomarin-rareconnections.com to find out how BioMarin RareConnections can help you.

What does this really mean?

When you connect with BioMarin RareConnections, you will be introduced to your Case Manager. Your BioMarin RareConnections Case Manager will help you coordinate all of the details of your treatment, including confirming that your insurance will pay for NAGLAZYME. Most insurance plans will provide coverage for NAGLAZYME, but if you need help getting insurance, your BioMarin RareConnections Case Manager will look for options to get you sufficient coverage. Your Case Manager will also put you in touch with other people at BioMarin who can help you work with your healthcare team and who can provide NAGLAZYME or MPS VI education to you and your family.

You can contact BioMarin RareConnections at 1‐866‐906‐6100, or by emailing support@biomarin-rareconnections.com.

References:

  1. NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.
  2. Giugliani R, Lampe C, Guffon N, et al. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome): 10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014;164A(8):1953-1964.
  3. Harmatz P, Giugliani R, Schwartz IV, et al. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine-4-sulfatase. Mol Genet Metab. 2008;94(4):469-475.
  4. Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019;14:118.
  5. Sweidler S, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet. 2005;134:144-151.

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.

Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

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.  

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.

Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

INDICATION

NAGLAZYME® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stairclimbing capacity.