laronidase

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

What is laronidase?

Laronidase 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.

Laronidase is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis) 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.

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

What should I discuss with my healthcare provider before receiving laronidase?

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.

How is laronidase given?

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

Laronidase is usually given once per week.

Laronidase must be given slowly, and the 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 to make sure you do not have an 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 laronidase is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while receiving laronidase?

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

What are the possible side effects of laronidase?

• 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 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.

What other drugs will affect laronidase?

Other drugs may affect 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.

Where can I get more information?

Your doctor or pharmacist can provide more information about laronidase.