Pronunciation:

gal-SUL-fase

Brand Names:

• Naglazyme

Dosage Forms:

• Solution

Warnings:

Warning: Hypersensitivity Reactions Including AnaphylaxisPatients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.Initiate galsulfase in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue galsulfase and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur .

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Enzyme

Uses of This Medicine:

Galsulfase injection is used to treat symptoms of an inherited disease called mucopolysaccharidosis (MPS VI) disease or Maroteaux-Lamy syndrome. This medicine improves walking and stair-climbing ability in patients who are lacking a certain enzyme called N-acetylgalactosamine 4-sulfatase in the body.

This medicine is to be given only by or under the direct supervision of a doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of galsulfase injection in children 5 years of age and older. Safety and efficacy have been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of galsulfase injection in the geriatric population. Safety and efficacy have not been established.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Fluid volume overload (increased volume of fluid in the body) or
• Heart disease or
• Lung disease or breathing problems—Use with caution. May increase risk for serious side effects.

• Sleep apnea—Use with caution. May make this condition worse.

Proper Use of This Medicine:

A nurse or other trained health professional will give you or your child this medicine in a hospital. This medicine is given through an IV catheter that is placed into one of your veins. This medicine must be given slowly, so the IV will remain in place for at least 4 hours. It is usually given once a week.

You or your child may also receive medicines (eg, allergy medicine, fever medicine) 30 to 60 minutes before starting treatment with this medicine.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress closely while receiving this medicine to make sure that it is working properly. Blood and urine tests may be needed to check for unwanted effects.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you or your child have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, skin rash, trouble breathing, or unusual tiredness or weakness.

Check with your doctor right away if you have bloody or cloudy urine, high blood pressure, or swelling of the face, feet, or lower legs. These maybe symptoms of serious kidney problem.

This medicine may cause infusion-related reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child start to have a fever, chills or shaking, chest pain, dizziness, flushing, headache, trouble breathing, hives, itching, or skin rash, lightheadedness, fainting, fast, pounding, or uneven heartbeat, or trouble breathing after receiving this medicine.

Tell your doctor right away if you have back pain, paralysis of the limbs, or loss of bladder or bowel control after receiving this medicine. These could be symptoms of a condition called spinal or cervical cord compression (SCC).

Your doctor may want you or your child to join a patient registry for patients using this medicine. This will help you monitor the progress of your disease while on long-term treatment using this medicine.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

Less common

Blurred or decreased vision

chest pain or tightness

difficult or labored breathing

dizziness

headache

hernia of the naval

nervousness

pounding in the ears

slow or fast heartbeat

swelling of the face

Incidence not known

Back pain

black, tarry stools

bleeding gums

bloody or cloudy urine

bluish lips or skin

chills

confusion

cough

difficulty swallowing

dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position

fever

hives, itching, skin rash

joint pain

large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs

loss of bladder control

loss of bowel control

nausea or vomiting

paralysis of the limbs

pinpoint red spots on the skin

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

stomach pain

swelling of the face, feet or lower legs

sweating

trouble breathing

unusual bleeding or bruising

unusual tiredness or weakness

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Diarrhea

ear pain

loss of appetite

Less common

Body aches or pain

burning, dry, or itching eyes

congestion

dryness or soreness of the throat

excessive tearing

loss of or increase in reflexes

runny or stuffy nose

tender, swollen glands in the neck

voice changes

Incidence not known

Burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings

difficulty with moving

loss of voice

muscle pain or stiffness

sneezing

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.