Sebelipase alfa

Sebelipase alfa is a replacement for an enzyme that the body usually makes to help break down fats and prevent them from accumulating in your cells.

Sebelipase alfa is used to treat lysosomal acid lipase deficiency. This is a serious genetic disorder that can harm important organs in the body and can cause early death. Sebelipase alfa provides the enzyme that the body is lacking.

Sebelipase alfa may also be used for reasons not mentioned in this medication guide.

Sebelipase alfa is given through an IV into a vein. A healthcare provider will administer this injection.

Sebelipase alfa is typically given once every other week. The IV infusion should be done slowly and may take 1 to 2 hours to finish.

You will be monitored closely during and after your sebelipase alfa injection to ensure you do not have an allergic reaction to the medication.

Inform your doctor if you notice any changes in your weight. Doses of sebelipase alfa are based on weight (especially in children), and any changes could affect the dose.

Follow all instructions on your medicine label and package. Inform all your healthcare providers about your medical conditions, allergies, and all the medicines you are taking.

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.

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.

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 weekComments:-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 weekComments:-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 weekComments:-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

Call your doctor for instructions if you miss an appointment for your Kanuma 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.

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