Vimizim contains an enzyme that occurs naturally in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Elosulfase alfa helps replace this missing enzyme in such people.

Vimizim is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis IV (4) type A, or MPS IV A, also called Morquio A syndrome.

MPS IVA is a metabolic disorder in which the body lacks the enzyme needed to break down certain natural substances. These substances can build up in the body, causing enlarged organs, abnormal bone structure, changes in facial features, breathing problems, heart problems, vision or hearing loss, and changes in mental or physical abilities.

Vimizim may improve walking and stair-climbing ability in people with this condition. However, this medication is not a cure for MPS IVA.

An allergic reaction may occur during or shortly after infusion of Vimizim. Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as chest discomfort, cough, trouble breathing, vomiting, skin rash or redness, pale skin, blue lips or fingernails, and feeling like you might pass out.

You may be more likely to have a reaction to Vimizim if you have a breathing disorder, or if you are sick with a fever at the time of your infusion.

Your breathing, blood pressure, and other vital signs will be watched closely during and after the infusion, so your caregivers can quickly treat any serious side effects that occur.

Before taking this medicine

Your Vimizim infusion may be delayed if you have a fever or cold symptoms.

Tell your doctor if you have ever had:

• breathing problems; or

• sleep apnea, and you use a continuous positive airway pressure (CPAP) machine.

Tell your doctor if you are pregnant or breastfeeding.

It is not known whether elosulfase alf will harm an unborn baby. However, MPS symptoms can get worse during pregnancy and may cause medical problems in both mother and baby. The benefit of treating MPS may outweigh any risks to the baby.

Your name may need to be listed on a Morquio A Registry while you are using this medicine. The purpose of this registry is to track the progression of this disorder and the effects that elosulfase alfa has on long-term treatment of Morquio A syndrome. The registry also tracks the effects of elosulfase alfa on a baby if you are pregnant or breastfeeding.

Vimizim is given as an infusion into a vein. A healthcare provider will give you this injection.

The infusion must be given slowly, and can take up to 4 hours or longer to complete.

Vimizim is usually given once per week. Follow your doctor's instructions.

Vimizim doses are based on weight (especially in children and teenagers). Your dose needs may change if you gain or lose weight.

About 30 to 60 minutes before each injection, you will be given other medications to help prevent a serious allergic reaction.

You will be watched closely after receiving Vimizim, to make sure you do not have an allergic reaction.

Usual Adult Dose for Mucopolysaccharidosis Type IV:

2 mg/kg via IV infusion once a week

Comments:
-Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.
-See Other Comments/Administration Advice for recommended dilution volume and infusion rates.

Use: For patients with mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome)

Usual Pediatric Dose for Mucopolysaccharidosis Type IV:

5 years or older: 2 mg/kg via IV infusion once a week

Comments:
-Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.
-See Other Comments/Administration Advice for recommended dilution volume and infusion rates.

Use: For patients with mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome)

Your Vimizim infusion may be delayed if you have a fever or cold symptoms.

Tell your doctor if you have ever had:

• breathing problems; or

• sleep apnea, and you use a continuous positive airway pressure (CPAP) machine.

Tell your doctor if you are pregnant or breastfeeding.

It is not known whether elosulfase alf will harm an unborn baby. However, MPS symptoms can get worse during pregnancy and may cause medical problems in both mother and baby. The benefit of treating MPS may outweigh any risks to the baby.

Your name may need to be listed on a Morquio A Registry while you are using this medicine. The purpose of this registry is to track the progression of this disorder and the effects that elosulfase alfa has on long-term treatment of Morquio A syndrome. The registry also tracks the effects of elosulfase alfa on a baby if you are pregnant or breastfeeding.

Call your doctor for instructions if you miss an appointment for your Vimizim injection.

Since this medication is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction to Vimizim: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

An allergic reaction may occur during or shortly after infusion of this medicine (up to 3 hours later). Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as:

• feeling like you might pass out, even while lying down;

• cough, chest discomfort, feeling short of breath;

• skin rash or redness, sudden warmth or tingly feeling;

• nausea, vomiting;

• pale skin; or

• blue lips or fingernails.

Common Vimizim side effects may include:

• nausea, vomiting, stomach pain;

• headache;

• fever, chills; or

• feeling tired.

Even though it may not be a side effect of Vimizim, increased pressure on the spinal cord is a complication of MPS IVA that may occur while you are using this medicine. Tell your doctor right away if you have any symptoms of spinal cord compression:

• back pain;

• loss of movement in any part of your body; or

• loss of bowel or bladder control.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.