WELCOME TO THE HOME INFUSION EXPERIENCE AN INFORMATION GUIDE FOR PEOPLE WITH MORQUIO A Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM (elosulfase alfa) infusions. Typical signs of anaphylaxis 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. Contact your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring. Please see Important Safety Information, including important warning, on back cover.
YOU ARE ON YOUR WAY TO MAKING THE TRANSITION TO HOME INFUSION This is an exciting time your doctor has determined that you can receive your VIMIZIM (elosulfase alfa) infusions at home. It will still be important for you to have regular visits with your physician, but getting your infusions at home means you won t have to spend time traveling to your clinic each week. You ll also be able to receive your treatment in the comfort of familiar surroundings. While home infusion is not an option for every person receiving VIMIZIM, your treatment team has determined that you are ready to start receiving your treatments at home. The goal of home infusion is to make your weekly infusion more convenient for you. In order for home infusion to be successful in the long-term, it is important that you understand your rights and your responsibilities. On the following pages, you will learn how to safely store VIMIZIM vials and supplies for use on your infusion day. You will also learn what you can expect from the infusion nurse that will be caring for you in your home environment. BioMarin will help you coordinate your home infusions. If you have any questions about your role, or the role of the home infusion nurse, please ask your doctor or your BioMarin Patient and Physician Support (BPPS) case manager.
GET ANSWERS TO YOUR QUESTIONS ABOUT HOME INFUSION What will change if I receive VIMIZIM (elosulfase alfa) infusions at home? The most significant difference with home infusion is that you will receive the shipments of VIMIZIM (elosulfase alfa) vials directly from a specialty pharmacy, and you will need to store them in your refrigerator. You will also work with your home infusion nurse to make sure that you have all of the supplies you need for each weekly infusion. What will stay the same if I receive my VIMIZIM infusions at home? Although there are some important differences between receiving your VIMIZIM infusions at home and in a clinic, many things will stay the same. The nurse who comes to your home on the scheduled day will prepare your medication, perform the infusion, and monitor your vital signs throughout just like when you receive your infusions in a clinic. Just as it was important not to miss any treatments at your clinic, the same is true for home infusions. If you need to reschedule one of your weekly appointments, you should call your nurse directly. If you are not able to reach your nurse, you should call your BPPS case manager to help you. Please see Important Safety Information, including important warning, on back cover. 3
PREPARING FOR YOUR FIRST HOME INFUSION RECEIVING AND STORING YOUR VIMIZIM (elosulfase alfa) Schedule with your case manager when the specialty pharmacy should deliver your VIMIZIM (elosulfase alfa). When scheduling your VIMIZIM delivery, make sure it is at a convenient time for you because you will need to be home to receive it. You should refrigerate VIMIZIM as soon as you get it. Store it in a separate container and keep it away from food Place a thermometer in your refrigerator so that you can monitor the temperature You should also follow a few simple rules when handling VIMIZIM. Never shake VIMIZIM vials Never freeze VIMIZIM Never heat VIMIZIM with hot water or a microwave Never use medicine that has been unrefrigerated for more than 24 hours NOTE: If your VIMIZIM has been heated, frozen, left unrefrigerated for more than 24 hours, or damaged in any way, separate the vials in question and tell your nurse, and contact BPPS immediately at 1-855-MORQUIO (1-855-667-7846). RECEIVING AND STORING YOUR INFUSION SUPPLIES Before your infusion supplies are delivered, find a convenient, cool, dry space to store them. If you store supplies in your kitchen cabinets, be sure the shelf is not close to a heat source, such as a water heater, stove, or refrigerator fan Your supply shipments will be delivered directly to your home and will include boxes of infusion sets and filters, and saline solution NOTE: You should work with your nurse to make sure that you are ready when the specialty pharmacy calls you each month to take your order to refill your VIMIZIM prescription and infusion supplies. 4
YOUR HOME INFUSION NURSE IS ON YOUR TEAM Creating a successful home infusion experience requires equal participation from you and from your nurse. Just as you have responsibilities, your nurse does, too. The nurse that you work with is expected to be prompt and professional at each home infusion appointment. He or she will come prepared to answer any of your questions about the infusion, and will be familiar with your doctor s orders. If your nurse is unable to come to any particular appointment, a trained backup nurse will come in their place and should be up to speed on everything they need to know about you and your treatment. Your nurse will be trained and ready to look for the signs of an allergic or other adverse reaction and will teach you how to recognize some of those signs yourself. He or she will also know when to call a doctor to seek additional medical attention if needed. Open communication is an essential part of good medical care. You should always feel like you can talk to your nurse about concerns or questions you may have related to your treatment. If you do not feel comfortable talking with your nurse for any reason, you should inform your doctor or BPPS case manager. Please see Important Safety Information, including important warning, on back cover. 5
STEPS FOR INFUSION DAY 1 Prepare yourself for your infusion. Be sure you are ready when your nurse arrives so that your infusion can be as efficient as possible. To help prevent allergic or adverse reactions during or after infusion, your doctor may recommend that you take medicine beforehand. If you use supplemental oxygen or continuous positive airway pressure when you sleep, be sure to have those treatments readily available during your infusion. It is important to be hydrated for the infusion, and you may also eat before the treatment. 2 Upon arrival, your nurse will check for several things before he or she begins the infusion: Your vital signs to make sure you don t have a fever or infection; be sure to tell your nurse about any illnesses or changes in your health; these changes may affect whether you should delay your weekly infusion The infusion supplies to make sure everything needed for the infusion is available, including emergency medications in the event of an allergic or adverse reaction 3 Your nurse will start an IV, prepare the VIMIZIM (elosulfase alfa), and begin the infusion. VIMIZIM is infused slowly, over a minimum range of 3.5 to 4.5 hours. During the infusion period, your nurse will monitor your vital signs. Your nurse will also periodically adjust your infusion rate. You will have some mobility during your infusion, but be careful not to disrupt your IV line. The antihistamines you receive may make you drowsy. Until you know how the medication affects you and how quickly it wears off, you should avoid driving, using heavy machinery, or doing anything else that could be dangerous if you are not alert. 4 After the infusion, your nurse will remove the IV line and carefully dispose of the needles, tubing, and any empty vials. Your nurse may wait with you for a short period of time after the infusion to watch for any signs of delayed allergic reactions. 6
IMPORTANT SAFETY INFORMATION FOR HOME INFUSIONS Talk to your doctor and your home infusion nurse to develop a plan everyone is comfortable with to manage allergic or adverse reactions. Signs of an allergic or adverse reaction include Cough Rash Throat tightness Hives Flushing Changes in skin color Low blood pressure Shortness of breath Chest pain Gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting If you experience any of these side effects after your nurse has left, call your doctor immediately or seek medical attention. Protecting VIMIZIM (elosulfase alfa) during power outages If there is an electrical outage in your home, avoid opening the door of the refrigerator until power is restored. You should also write down the time when the power outage began. As soon as the electricity comes back on, write down the time and the temperature inside the refrigerator. For power outages lasting longer than 24 hours, you should move VIMIZIM to a location that has refrigeration. Follow these steps: Remove VIMIZIM from the refrigerator; write down the time and temperature inside the refrigerator when you remove the medication Place VIMIZIM in an insulated container, such as a small ice chest with a lid; there is no need to add ice or coolant Transport the insulated container to the new location and place VIMIZIM in the new refrigerator Write down the time and the internal temperature of the new refrigerator Now that you have read through this guide, you have taken an important step to prepare for your first home infusion. If you have any questions before your first scheduled appointment, be sure to contact your doctor or BPPS case manager. Please see Important Safety Information, including important warning, on back cover. 7
INDICATION VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). IMPORTANT SAFETY INFORMATION Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM (elosulfase alfa) infusions. Typical signs of anaphylaxis 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. Contact your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring. VIMIZIM is a prescription medicine. Before treatment with VIMIZIM, it is important to discuss your medical history with your doctor. Tell your doctor if you are sick or taking any medication and if you are allergic to any medicines. Also tell your doctor if you are pregnant, planning to become pregnant, or are a nursing mother. Your doctor will decide if VIMIZIM is right for you. If you have questions or would like more information about VIMIZIM, contact your doctor. Anaphylaxis can occur during any VIMIZIM infusion and up to three hours after any infusion, and hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as six days after infusion. Serious and severe reactions can happen with VIMIZIM treatment, including life-threatening allergic reactions (anaphylaxis), hives, swelling, cough, shortness of breath, and flushing. You should receive medication such as antihistamines before VIMIZIM infusions to reduce the risk of reactions. If a reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. If a severe reaction occurs, the infusion should be stopped immediately and you will receive appropriate medical treatment. If you have acute febrile or respiratory illness at the time of VIMIZIM infusion you may be at higher risk of lifethreatening complications from hypersensitivity reactions. If you use supplemental oxygen or continuous positive airway pressure (CPAP) you should have it available during your infusion in the event of a sudden reaction, or extreme drowsiness/sleep from antihistamines. Spinal cord damage may occur due to the natural MPS IVA disease process. Signs of spinal cord injury include back pain, numbness and paralysis, and loss of bladder and bowel control. Contact your doctor immediately if you develop any of these symptoms. The most common side effects reported during VIMIZIM infusions included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. These are not all of the possible side effects with VIMIZIM. Talk to your doctor if you have any symptoms that bother you or that do not go away. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. For more information, call BioMarin Patient and Physician Support (BPPS) at 1-855-MORQUIO (1-855-667-7846). Please see accompanying full Prescribing Information, including important warning, or visit www.vimizim.com. 2014 BioMarin Pharmaceutical Inc. All Rights Reserved. USMPS0666ENJUL2014