Cerezyme

Cerezyme is used to treat non-central nervous system (CNS) manifestations of Type I or Type 3 Gaucher disease in adults and children. It is a synthetic form of the enzyme glucocerebrosidase that is deficient or absent in Gaucher disease patients, and is given by intravenous infusion.

Gaucher disease is a genetic condition in which the body lacks the glucocerebrosidase enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured. Cerezyme is an enzyme replacement therapy for people with Gaucher's.

Cerezyme may improve the condition of the liver, spleen, bones, and blood cells in people with Type I or Type 3 Gaucher disease. However, this medicine is not a cure for this condition.

There is a Gaucher Patient Registry that tracks the variability and progression of Gaucher's disease and the long-term treatment effects of Cerezyme. Ask your healthcare provider for more information. Although participation in this registry is voluntary and may involve long-term follow-up, you are encouraged to enrol. For information visit www.registrynxt.com or call 1-800- 745-4447, extension 15500.

Cerezyme (imiglucerase) gained FDA approval on May 23, 1994. There is no generic or biosimilar.

Do not receive Cerezyme if you are allergic to imiglucerase, Cerezyme, or any of the inactive ingredients in the injection vial.

Before you receive Cerezyme, tell your healthcare provider about all your medical conditions, including if you:

• Have a breathing problem such as pneumonia or pulmonary hypertension
• Are pregnant or intend to become pregnant
• Are breastfeeding or intend to breastfeed.

Pregnancy

It is not known if Cerezyme will harm an unborn baby. Tell your healthcare provider if you are pregnant or plan to become pregnant while using this medicine. There is a pregnancy sub-registry that monitors the effects of Cerezyme on pregnant women and their offspring.

Breastfeeding

It is not known whether Cerezyme passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breastfeeding.

You will receive Cerezyme as an intravenous infusion. A healthcare provider will give you this injection.

• Cerezyme is usually given every 2 weeks, but you may need the medicine more often when you first start using it. Follow your doctor's dosing instructions very carefully.
• You may be given other medicines to prevent certain side effects of Cerezyme. Take these medicines exactly as directed.

Tell your doctor if you have any weight changes. The dose of Cerezyme is based on your weight.

Warnings

Cerezyme carries a Boxed Warning for hypersensitivity reactions, including anaphylaxis.

Risk of Life-Threatening Hypersensitivity Reactions

Life-threatening hypersensitivity reactions, including anaphylaxis, and infusion-associated reactions (IAR) may occur with Cerezyme treatment. These may occur during the early course of enzyme replacement therapy and after an extended duration of therapy. Seek emergency medical attention if you experience any symptoms of an allergic reaction, including difficulty breathing, closing of the throat, hives, itching, flushing, dizziness or fainting, or swelling of the lips, tongue, or face, have occurred with Cerezyme.

Common Side Effects

The most common side effects of Cerezyme are:

• Back pain
• Chills
• Dizziness
• Fatigue
• Headache
• Hypersensitivity reactions
• Nausea
• Fever
• Vomiting.

Notes:

To report suspected adverse reactions, contact Genzyme at 1-800-633-1610 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Usual Adult Dose of Cerezyme for Gaucher Disease

• Initial dosage range: 2.5 units/kg intravenously 3 times a week to 60 units/kg once every 2 weeks.
• The dose will be titrated based on your disease severity and therapeutic goals.
• Cerezyme is diluted before use and infused over 1 to 2 hours.
• If a severe IAR occurs, discontinue immediately and begin appropriate medical treatment, including epinephrine.

Usual Pediatric Dose of Cerezyme for Gaucher Disease

• Initial dosage: 2.5 units/kg intravenously 3 times a week to 60 units/kg once every 2 weeks.
• Dilute to the suggested infusion volume depending on weight and infuse over 1 to 2 hours as directed by the prescribing information.
• If a severe IAR occurs, discontinue immediately and begin appropriate medical treatment, including epinephrine.

Call your doctor for instructions if you miss a dose of Cerezyme.

Other drugs may interact with Cerezyme, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your health care providers about all medicines you use now and any medicine you start or stop using.

Active ingredient: imiglucerase 424 units (delivers 400 units)

Inactive ingredients: mannitol (340 mg), polysorbate 80, NF (1.06 mg), and sodium citrates: disodium hydrogen citrate (36 mg) and trisodium citrate (104 mg).

Supplied as a lyophilized powder in a single-dose vial.

Manufactured by Genzyme Corporation, Cambridge, MA 02141; A Sanofi company U.S. License Number: 1596