galsulfase

What is the most important information I should know about galsulfase?

What is galsulfase?

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

Galsulfase is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis VI (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis type 6), or MPS VI, also called Maroteaux-Lamy syndrome.

MPS VI 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.

Galsulfase may also be used for purposes not listed in this medication guide.

What should I discuss with my health care provider before receiving galsulfase?

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.

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

How is galsulfase given?

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

Galsulfase is usually given once per week.

Galsulfase must be given slowly, and the infusion can take up to 4 hours to complete.

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

Your doctor will need to check your progress on a regular basis.

What happens if I miss a dose?

What happens if I overdose?

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

What should I avoid while receiving galsulfase?

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

What are the possible side effects of galsulfase?

• rapid breathing, or feeling short of breath;
• chest pain;
• feeling like you might pass out, even while lying down;
• fever, chills;
• skin rash or redness, itching;
• eye redness;
• headache; or
• vomiting, stomach pain.

Even though it may not be a side effect of galsulfase, increased pressure on the spinal cord is a complication of MPS VI that may occur while you are using galsulfase. 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, loss of bowel or bladder control.

• snoring or sleep apnea, trouble breathing;
• swelling, rapid weight gain; or
• shortness of breath (even with mild exertion).

Common side effects may include:

• fever, chills;
• trouble breathing;
• pain;
• rash or itching;
• headache; or
• nausea, vomiting, stomach pain.

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.

What other drugs will affect galsulfase?

Other drugs may affect galsulfase, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Where can I get more information?

Your doctor or pharmacist can provide more information about galsulfase.