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.

Bones? Heart? Lungs? How are they related?

Morquio A (also called MPS IVA) is an inherited condition that affects major organ systems in the body. People born with Morquio A are missing or don’t make enough of an important enzyme and, as a result, they can’t break down certain materials in the body. These materials eventually build up in tissues, bones, and major organ systems. Morquio A can cause potentially serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death.

The more you know about how Morquio A works and what parts of your body it affects, the more power you will have to be your own advocate.

The menu options to the left will help you find out more about Morquio A and the recently published “International Guidelines for the Management and Treatment of Morquio A Syndrome.” Here is what you will find in each section:

What is Morquio A? Find the answer to this question with facts about how Morquio A works. Take a look at this section to understand the science behind Morquio A.

Learn about the early signs of Morquio A, and see the full list of symptoms that go along with the syndrome.

This section is full of information about how Morquio A is diagnosed. Since Morquio A can progress over time, it is important to talk to your doctor about getting a diagnosis as early as possible to prevent more damage to different parts of your body.

Now that you know about Morquio A, find out how VIMIZIM® (elosulfase alfa) enzyme replacement therapy works.

Download your own copy of the recently published Guidelines and learn how they can help you manage your Morquio A.

Do you have questions about treating Morquio A with VIMIZIM?

Talk to BioMarin RareConnections™

Need a geneticist? Find one with experience in Morquio A near you.

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