IMPORTANT SAFETY INFORMATION
WARNING: Life-threatening allergic reactions have occurred in some patients during VIMIZIM® (elosulfase alfa) infusions and up to 3 hours after infusion. Patients with acute respiratory illness may be at increased risk and require additional monitoring.

GLOSSARY

Anaphylaxis - A serious allergic reaction. Symptoms of anaphylaxis can include itchy throat, rash, low blood pressure, or throat swelling.
BioMarin RareConnections™ - BioMarin RareConnections is dedicated to getting you access to VIMIZIM® (elosulfase alfa), and helping you coordinate all the details of your treatment, as well as providing you with other resources to help you manage Morquio A. If you’re interested in this service, give us a call at 1-866-906-6100.
Carrier - A person who has a genetic mutation, but does not show symptoms of a disease. However, they can pass this mutation on to their children.
Cells - Cells are called the “building blocks of life” because they are the substance that makes up all living things. In fact, the human body is made up of 100 trillion cells.
Clinical studies - Scientists use these tests to find out the safety and effectiveness of a medicine.
Endurance - A measure of how long a person can push himself or herself physically. Endurance measures how well your whole body performs—from heart and lungs to bones and muscles.
Enzyme - A protein that performs a specific job in the body.
Enzyme replacement therapy (ERT) - This medical treatment replaces deficient enzymes.
Extension study - A continuation of a study or studies performed by scientists to learn how well a medicine works.
Glycosaminoglycans (GAGs) - Long, sugar carbohydrate chains. Certain GAGs can’t be broken down by the body in someone with Morquio A.
GALNS - People with Morquio A lack the N-acetylgalactosamine-6 sulfatase (GALNS) enzyme, which works to break down GAGs.
Hypersensitivity - Also called a hypersensitivity reaction. This occurs when the body has an extreme response to an allergen or unfamiliar substance. Hypersensitivity reactions range from mild to serious, and they also range in how quickly they develop. Some reactions occur almost immediately, while others may not happen until after exposure to an allergen.
Keratan sulfate (KS) - A type of GAG found in the cornea, bone, and cartilage.
Lysosomal storage disorders - A group of rare, inherited diseases usually involving a malfunctioning enzyme.
Mucopolysaccharide - A kind of sticky molecule, also known as a GAG, that the body uses to build certain tissues. A healthy body continuously breaks down used mucopolysaccharide molecules and recycles them.
Mucopolysaccharidosis (MPS) - People with MPS disorders are not able to break down cellular material in a normal way because they lack the enzymes needed to do so. Morquio A is a kind of MPS disorder. Morquio A is also known as MPS IVA.
Placebo - A placebo is a substance that is made to look like a medication. But, unlike a real medication, placebos don’t contain any active ingredients.
Port - A device that is placed in the chest area or under the skin using local or general anesthetics. The advantage of a port is that it gives doctors and nurses easier access to veins.
Protein -A protein is a molecule that is made up of amino acids. Most enzymes are proteins. They have a lot of important jobs in the body, including helping to make important chemical reactions happen.
Protein-based - Made up of proteins.
Recessive - Traits that are often hidden by dominant traits.
Respiratory - Anything related to breathing.
Six-minute walk test - Also known as the 6MWT, this test measures how far a person can walk in 6 minutes, which is an indicator of endurance.
Tissues - A grouping of the same cell type.
Walking aids - Devices that help people walk, including canes, walkers, and wheelchairs.

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 life-threatening 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 RareConnections™ at 1-866-906-6100.

CONTACT US

BioMarin Pharmaceutical Inc.

Novato, CA 94949

BioMarin RareConnections™: 1-866-906-6100

Biomarin