Keeping your treatment on track

Enzyme replacement therapy (ERT) is lifelong. In order for you to benefit 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, GAG will again build up, and symptoms may return.

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

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

  • 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.

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

Learn about the transition from pediatric care to adult care

Read the answers to frequently asked questions about MPS VI and NAGLAZYME treatment

Benefits of consistent treatment

In MPS VI, glycosaminoglycan (GAG) buildup 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 GAG waste. It reduces and stabilizes GAG levels and may help to slow the decline associated with this condition.

Treatment tested over the long term

Long‐term studies have been done in patients taking NAGLAZYME. These studies examined walking, how well patients’ lungs worked, and GAG levels in patients who had been taking NAGLAZYME for 6 months to 10 years (the average time on NAGLAZYME therapy was 6.8 years).

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 GAG protein in the urine.

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.

The National Health Care Transition Center offers a helpful readiness assessment tool. Visit the center’s web site to learn more. Download this questionnaire and use it to discuss the transition process with your doctor.

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.

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 GAG that causes symptoms of MPS VI. Learn more

Could NAGLAZYME be right for me?

All patients with MPS VI can benefit from NAGLAZYME. Your doctor can tell you whether NAGLAZYME may be right for you. Learn more

What does the doctor need to know about me before I start treatment with NAGLAZYME?

Your doctor will need to do a physical exam. He or she may also need to know:

  • If you are pregnant, may become pregnant, or are breast‐feeding
  • Have medical problems such as trouble breathing, even if these are not related to MPS VI
  • Have allergies, even if they are not related to MPS VI
  • Take other medications (prescription or over‐the‐counter) or supplements or vitamins
How do I take NAGLAZYME?

NAGLAZYME is given by infusion. That means that once a week, you will go to a hospital or infusion center. At the center, a doctor or nurse will give you NAGLAZYME through an IV line. The IV gets the enzyme right into your bloodstream. The blood carries it to your body’s cells and organs to break down GAG. Learn more

How long does a NAGLAZYME infusion take?

The infusion itself takes about 4 hours. 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. 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 you need to be your best. 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. 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.

Severe and life‐threatening allergic reactions can occur during NAGLAZYME 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.

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.

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.

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.

How long will I have to take NAGLAZYME?

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

What if I miss a treatment?

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

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

What changes can I expect from NAGLAZYME?

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

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.