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.

Morquio A: it’s inherited and progressive.

Morquio A is a recessively inherited condition. That means that both parents must have the defective gene in order to risk passing Morquio A on to their children.

Our genes are what determine most of our physical characteristics. That includes things like our hair color, blood type, and eye color. Genes are also responsible for some kinds of health problems, such as Morquio A.

When 2 parents who are carriers have a child, there is a 25% chance (1 out of 4) that the child will have Morquio A

When 2 parents who are carriers have a child, there is a 25% chance (1 in 4) that the child will have Morquio A.

People who have Morquio A can’t make an important enzyme that is a key part of clearing out materials from cells. The name of this enzyme is N-acetylgalactosamine-6 sulfatase, or GALNS for short.

GALNS enzymes break down a type of cellular molecule called a glycosaminoglycan (GAG). When GAG molecules are not broken down, they build up in the parts of the cell called the lysosomes that are in charge of breaking down materials.

In people with Morquio A, certain GAGs build up in lysosomes of cells throughout the tissues and organs of the body. This buildup can cause potentially serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, and difficulty breathing. It can also limit your endurance, making it harder to move around and complete your daily activities.

A progressive condition gets worse over time. Morquio A is a progressive condition, which means that it gets worse as time goes on. The recently published “International Guidelines for the Management and Treatment of Morquio A Syndrome” highlight the importance of getting an early diagnosis to be able to start taking an enzyme replacement therapy as soon as possible. Spotting the signs and symptoms of Morquio A can help lead to a diagnosis.

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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.


BioMarin Pharmaceutical Inc.

Novato, CA 94949

BioMarin RareConnections™: 1-866-906-6100