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.

Recognizing the signs and symptoms of Morquio A

When glycosaminoglycans (GAGs) build up in the bodies of people with Morquio A, the results can potentially be severe. People with Morquio A have physical problems and organ complications that can lead to difficulties performing daily activities, and may cause them to need walking aids or wheelchairs.

The signs and symptoms of Morquio A are not usually easy to see at birth. By the age of 2 or 3, enough GAGs have built up in the child’s body to start causing noticeable problems.

The signs and symptoms of Morquio A can affect many different parts of your body.

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Skeletal symptoms
Nonskeletal symptoms
Skeletal deformities
Breathing problems
Short trunk
Heart valve problems
Weakness in the neck
Muscle weakness
Spinal cord compression
Eye problems
Knock knees
Hearing loss
Overly flexible joints
Dental problems
Abnormal walk
Enlarged liver
Spinal problems
Enlarged spleen
Chest deformities
Limited energy

According to the recently published “International Guidelines for the Management and Treatment of Morquio A Syndrome,” getting a diagnosis is important so that treatment with an enzyme replacement therapy can be started right away.

Visit the Morquio LIVE webinar series at morquiosity.com to watch videos that give more detail about how VIMIZIM works and whether or not it could be right for 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