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

Aldurazyme is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis or MPS I, also called Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome.

MPS I is a metabolic disorder in which the body lacks the enzyme needed to break down certain 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.

Aldurazyme may improve breathing and walking ability in people with this condition. However, this medication is not a cure for MPS I.

Before you receive Aldurazyme, tell your doctor if you have heart disease, kidney disease, lung disease, seizures, migraine headaches, or sleep apnea.

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

Your doctor may also prescribe other medications to help prevent an allergic reaction to Aldurazyme. Take all of your medications as directed.

Before taking this medicine

You should not be treated with Aldurazyme if you are allergic to laronidase. Your infusion may be delayed if you have a fever or cold symptoms.

To make sure Aldurazyme is safe for you, tell your doctor if you have ever had:

• breathing problems;

• heart disease; 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 laronidase 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 an MPS I Registry while you are using this medicine. The purpose of this registry is to track the progression of this disorder and the effects that laronidase has on long-term treatment of MPS I. The registry also tracks the effects of laronidase on a baby if you are pregnant or breastfeeding.

Aldurazyme is injected into a vein through an IV. A healthcare provider will give you this injection.

Aldurazyme 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 injection.

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

An hour before each injection, you will be given other medications to help prevent a serious allergic reaction.

After each injection, you will be watched closely for 60 minutes or longer to make sure you do not have an allergic reaction.

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

It may take up to several months to years before you get the full benefit of treatment with Aldurazyme. Keep receiving your injections as directed and tell your doctor if your symptoms do not improve or if they get worse.

Usual Adult Dose for Mucopolysaccharidosis Type I:

0.58 mg/kg IV once a week

Comments:
-Clinical studies did not include patients 65 years and older and it is therefore unknown if they respond differently than younger patients.

Uses: For the treatment of Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established

Usual Pediatric Dose for Mucopolysaccharidosis Type I:

6 months or older: 0.58 mg/kg IV once a week

Uses: For the treatment of Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established

You should not be treated with Aldurazyme if you are allergic to laronidase. Your infusion may be delayed if you have a fever or cold symptoms.

To make sure Aldurazyme is safe for you, tell your doctor if you have ever had:

• breathing problems;

• heart disease; 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 laronidase 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 an MPS I Registry while you are using this medicine. The purpose of this registry is to track the progression of this disorder and the effects that laronidase has on long-term treatment of MPS I. The registry also tracks the effects of laronidase on a baby if you are pregnant or breastfeeding.

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

Since Aldurazyme 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 Aldurazyme: 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:

• rapid breathing, or feeling short of breath;

• wheezing, chest tightness;

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

• fast or slow heartbeats;

• sweating, cold or clammy skin;

• pale skin; or

• blue lips or fingernails.

Common Aldurazyme side effects may include:

• fever, chills;

• cold symptoms such as stuffy nose, sneezing, sore throat;

• increased blood pressure;

• warmth, redness, numbness, or tingly feeling;

• fast heartbeats;

• overactive reflexes; or

• pain, swelling, burning, or irritation around the IV needle.

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.

Other drugs may interact with laronidase, 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.