Patient Information about NAGLAZYME Please read this information thoroughly before you begin therapy with NAGLAZYME® (galsulfase). This information should not replace discussions with your doctor about your therapy or your medical condition. Talk to your doctor about NAGLAZYME before you begin treatment and at your regular checkups, in case anything has changed. If you have any questions about NAGLAZYME, ask your doctor. Only your doctor can determine if NAGLAZYME is right for you. Please see full indication and important safety information on reverse side. What is NAGLAZYME? What is NAGLAZYME used for? Am I a candidate for NAGLAZYME? How does NAGLAZYME work? How is NAGLAZYME administered? How long does an infusion take? What happens if I miss an infusion? Will it hurt? How long will I need to take NAGLAZYME? How will I feel during my infusions? What does my doctor need to know about me before I start therapy with NAGLAZYME? Can I take NAGLAZYME with other medications? What are the possible side effects with NAGLAZYME? What changes can I expect from taking NAGLAZYME? Does BioMarin offer support programs and services? Am I eligible for the Clinical Surveillance Program? Where can I get more information about my disease and treatment options? Q: What is NAGLAZYME? A: NAGLAZYME® (galsulfase) belongs to a class of drugs called enzyme replacement therapies (or ERT) that provides people with sufficient quantities of an important enzyme that their own body cannot make. The main ingredient in NAGLAZYME is a highly purified protein that is identical to a naturally occurring form of the human enzyme arylsulfatase B (ASB). Q: What is NAGLAZYME used for? A: NAGLAZYME is prescribed for people with Maroteaux-Lamy syndrome, also called Mucopolysaccharidosis VI (pronounced mu-ko-pol-ee-sak-ah-ri-doh-sis six), or MPS VI. MPS VI is a rare inherited disorder caused by a deficiency of the enzyme ASB. Normally, this enzyme breaks down substances called glycosaminoglycans (GAGs). People with MPS VI produce little or no enzyme, so GAGs can build up in many tissues and organs of their body—including the skin, heart valves, airways, and skeleton–producing widespread abnormalities over time. Q: Am I a candidate for NAGLAZYME? A: All patients with MPS VI are candidates for NAGLAZYME. For more information, speak with your doctor. Q: How does NAGLAZYME work? A: Your body is missing an enzyme which breaks down GAGs. NAGLAZYME provides a source of the missing enzyme at levels that provide the enzyme activity needed to break down GAG buildup. It is important to talk to your doctor about receiving infusions on a regular schedule and monitoring your GAG levels. Q: How is NAGLAZYME administered? A: NAGLAZYME is given by intravenous (IV) infusion, meaning that the medication is delivered directly into your bloodstream, and from there to your body’s cells and organs to break down the GAG buildup. NAGLAZYME is administered by your physician at a hospital or an infusion center. Q: How long does an infusion take? A: An infusion of NAGLAZYME takes at least 4 hours, and is given weekly. Q: What happens if I miss an infusion? A: If you miss an infusion, talk to your doctor about rescheduling your next dose. Occasionally, patients miss a dose because of illness. Be sure to let your physician know if you have a fever or respiratory illness to determine if you should delay your infusion. Q: Will it hurt? A: Starting the IV for the infusion may cause some pain or discomfort. There are products available that can numb the skin and reduce this discomfort. Ask your doctor what is right for you. Q: How long will I need to take NAGLAZYME? A: MPS VI is a lifelong disease. Regular replacement of the missing ASB enzyme with NAGLAZYME helps keep NAGLAZYME working in your body. Q: How will I feel during my infusions? A: Some patients feel fine and use the time to play games or work on their laptops. Other patients may feel sleepy or tired during and after the infusions, possibly due to premedications. If this is a concern for you, talk to your doctor to find out if you are a candidate for medications that are less likely to make you sleepy. There is also the possibility that you won’t feel well due to adverse reactions as described below. If you start feeling badly during the infusion, it is important to let the medical staff know. Q: What does my doctor need to know about me before I start therapy with NAGLAZYME? A: As with beginning any new treatment, your doctor will need to evaluate you to determine whether NAGLAZYME is right for you. This evaluation may include a physical examination. Your doctor may also wish to know whether you: Are or plan to become pregnant Are breast-feeding Have or have had any medical problems, such as breathing problems, even if not related to MPS VI Have or have had any allergies, even if not related to MPS VI Take any other medications (prescription or over-the-counter) or dietary supplements Please contact your physician if you have any questions regarding your evaluation, or whether NAGLAZYME is right for you. Q: Can I take NAGLAZYME with other medications? A: Always discuss your medications with your doctor. Before you begin therapy with NAGLAZYME, be sure your doctor is aware of any medications you are currently taking, as well as any medications you have recently stopped taking. When people take 2 or more medications at the same time, there is always a possibility that they will interact. Make sure to tell your doctor about all prescriptions and over-the-counter medications and dietary supplements you are taking or have taken recently. Q: What are the possible side effects with NAGLAZYME? A: In clinical studies, the most common symptoms of NAGLAZYME infusion reactions included fever, chills, headache, rash, and mild to moderate hives. Nausea, vomiting, high blood pressure, chest pain, abdominal pain, malaise, and joint pain were also reported. Severe reactions included angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea, and urticaria. Ask your physician for more information about these symptoms. Most of these infusion-related reactions were managed by temporarily stopping the infusion, slowing the infusion rate, or by giving patients additional medications to treat the reaction. No patients discontinued NAGLAZYME treatment because of the reactions. Q: What changes can I expect from taking NAGLAZYME? A: In clinical studies, NAGLAZYME reduced the levels of GAGs in urine, evidence that NAGLAZYME was working. When starting NAGLAZYME treatment, you may not feel any different right away. With time, NAGLAZYME has been shown to improve endurance. You may be able to walk further or climb more stairs. You may feel stronger and not tire as easily. These improvements may be reflective of heart, lung, and joint function. It is important to continue receiving regular therapy with NAGLAZYME as directed by your doctor. Even though you may be feeling better, do not stop taking any medications without first discussing it with your doctor. Q: Does BioMarin offer support programs and services? A: BioMarin Patient and Physician Support (BPPS) is staffed by patient advocates with expertise in customer service, reimbursement, insurance, case management, and the healthcare delivery system. BPPS patient advocates provide individualized support to help guide each physician and patient through the treatment process. We can send you information about MPS VI and help you find healthcare professionals with expertise in MPS VI and treatment centers in your area. For information about educational resources, health insurance, and other programs, please contact BPPS at 866-906-6100 or bpps@bmrn.com or visit www.naglazyme.com. Q: Am I eligible for the Clinical Surveillance Program? Another program BioMarin has established is a Clinical Surveillance Program (CSP). The CSP is an ongoing observational database that tracks the natural history and outcomes for people with MPS VI, including those treated with NAGLAZYME. It is designed to help doctors and other health professionals better understand, treat, and manage MPS VI. Since MPS VI is a rare condition, the information collected on known individuals with MPS VI can help healthcare professionals learn from the collective experience of others. The goal is for all individuals with MPS VI to be part of the program. Patients are encouraged to participate whether or not they have received any therapy. Please contact your physician or BPPS at 866-906-6100 for more information. Q: Where can I get more information about my disease and treatment options? A: For additional information, please visit the following Websites: www.mpssociety.org www.mpsvi.com