Sebelipase alfa is a copy of an enzyme that is normally produced in the body to help break down fats and keep them from building up in your cells.

Sebelipase alfa is used to treat lysosomal acid lipase deficiency. This is a life-threatening genetic disorder that can damage major organs in the body and lead to early death. Sebelipase alfa replaces the missing enzyme when the body does not have enough of its own.

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

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

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

Sebelipase alfa is usually given once every other week. The IV infusion must be given slowly and can take 1 to 2 hours to complete.

You will be watched closely during and after receiving sebelipase alfa, to make sure you do not have an allergic reaction to the medication.

Tell your doctor if you have any changes in weight. Sebelipase alfa doses are based on weight (especially in children), and any changes may affect the dose.

Usual Adult Dose for Wolman's Disease:

-Recommended dosage: 1 mg/kg as an IV infusion every other week
-Patients with suboptimal clinical response:
---Increase the dosage to 3 mg/kg IV every other week

Comments:
-Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers such as alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or parameters of lipid metabolism such as low-density lipoprotein cholesterol (LDL-c), or triglycerides (TG).

Use: For the treatment of lysosomal acid lipase (LAL) deficiency

Usual Pediatric Dose for Wolman's Disease:

Infants with Rapidly Progressive LAL Deficiency within the first 6 months of life:
-Recommended dosage: 1 mg/kg IV once a week
-Patients with a suboptimal clinical response:
---Increase dosage to 3 mg/kg IV once a week
-Patients with continued suboptimal clinical response:
---Increase dosage to 5 mg/kg once a week

Comments:
-Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly.

Pediatric patients over 6 months of age:
-Recommended dosage: 1 mg/kg as an IV infusion every other week
-Patients with suboptimal clinical response:
---Increase the dosage to 3 mg/kg IV every other week

Comments:
-Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers such as alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or parameters of lipid metabolism such as low-density lipoprotein cholesterol (LDL-c), or triglycerides (TG).

Use: For the treatment of lysosomal acid lipase (LAL) deficiency

You should not be treated with sebelipase alfa if you are allergic to it.

To make sure sebelipase alfa is safe for you, tell your doctor if you have:

• an allergy to eggs or egg products.

It is not known whether sebelipase alfa will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

It is not known whether sebelipase alfa passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

Sebelipase alfa is not approved for use by anyone younger than 1 month old.

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

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

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: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may occur during the injection. Tell your caregiver right away if you feel itchy, flushed, chilled or feverish, or if you have a runny nose, eye irritation, diarrhea, chest discomfort, or trouble breathing.

Sebelipase alfa may cause serious side effects. Call your doctor at once if you have:

• severe skin rash, itching, or swelling;

• agitation, irritability;

• stomach pain; or

• low red blood cells (anemia)--pale skin, feeling light-headed or short of breath, rapid heart rate, trouble concentrating.

Common side effects of sebelipase alfa may include:

• fever, weakness;

• runny nose, sinus pain, sore throat, cough;

• rash;

• diarrhea, constipation;

• nausea, vomiting; or

• headache.

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.