Galsulfase

Galsulfase contains an enzyme that is normally found in the body of healthy individuals. Some people do not have this enzyme due to a genetic disorder. Galsulfase helps to replace the missing enzyme in these individuals.

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

MPS VI is a metabolic disorder where the body does not produce the enzyme needed to break down certain natural substances. These substances can accumulate in the body, leading to enlarged organs, abnormal bone structure, changes in facial features, breathing difficulties, heart problems, vision or hearing loss, and alterations in mental or physical abilities.

Galsulfase may enhance walking and stair-climbing abilities in individuals with this condition. However, this medication is not a cure for MPS VI.

Galsulfase may also be used for reasons not mentioned in this medication guide.

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

Galsulfase is usually given once per week.

Tell your doctor if you have been sick with a fever or cold. You may need to wait until you get better before receiving your dose of galsulfase.

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.

An allergic reaction may happen during or soon after the infusion of this medicine. Inform your caregivers or seek emergency medical help immediately if you notice any signs of a severe allergic reaction, such as chest pain, difficulty breathing, vomiting, skin rash or redness, and feeling faint.

Your galsulfase infusion might be postponed if you have a fever or cold symptoms.

Let your doctor know if you have ever experienced:

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

Inform your doctor if you are pregnant or breastfeeding.

Your name may need to be included in an MPS IV Registry while you are receiving galsulfase. This registry is meant to monitor the progression of this disorder and the impact that galsulfase has on long-term treatment of MPS IV. The registry also keeps track of the effects of galsulfase on a baby if you are pregnant or breastfeeding.

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

An allergic reaction may happen during or shortly after the infusion of galsulfase (up to 24 hours later). Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as:

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

Galsulfase may cause serious side effects. Call your doctor immediately if you have:

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

Common side effects of galsulfase may include:

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

Usual Adult Dose for Mucopolysaccharidosis Type VI:

1 mg/kg IV once a weekComments:-Clinical studies did not include patients older than 29 years and it is therefore unknown if they respond differently than younger patients.Use: For the treatment of patients with Mucopolysaccharidosis VI (MPS VI [Maroteaux-Lamy syndrome])

Usual Pediatric Dose for Mucopolysaccharidosis Type VI:

5 years or older: 1 mg/kg IV once a weekUse: For the treatment of patients with MPS VI (Maroteaux-Lamy syndrome)

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

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

Follow your doctor's instructions regarding any limits on food, drinks, or activities.

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.