Sebelipase alfa

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.

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

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

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of sebelipase alfa, there are no specific foods that you must exclude from your diet when receiving this medication.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if sebelipase alfa crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using sebelipase alfa.

The dose your doctor recommends may be based on the following:

• your weight
• the condition being treated
• how you respond to this medication

Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life

The recommended dose of Kanuma (sebelipase alfa) for infants less than 6 months old and who have a quickly progressing disease is 1 mg/kg given once weekly. This dose can be increased to a maximum of 3 mg/kg weekly for patients who do not achieve the desired response at the lower dose.

Pediatric and Adult Patients with LAL Deficiency

The recommended dose for children and adults with LAL deficiency is 1 mg/kg given once every other week.

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

Lysosomal Acid Lipase Deficiency

Treatment of lysosomal acid lipase deficiency1 7 (designated an orphan drug by FDA for this use).2

Survival benefit demonstrated in patients with rapidly progressive infantile-onset disease (i.e., Wolman disease).1 8 9

In pediatric patients and adults with late-onset disease (i.e., cholesterol ester storage disease), improvements in disease-related lipid and hepatic abnormalities demonstrated.1 7

Effects on cardiovascular morbidity and mortality and progression of liver disease not established.1 7

Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Use sebelipase alfa as ordered by your doctor. Read all information given to you. Follow all instructions closely.

• It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

• Call your doctor to find out what to do.

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Kanuma: 20 mg/10 mL (10 mL) [contains albumin human, egg white (egg protein)]

• Enzyme, Replacement Therapy

LAL deficiency: Refer to adult dosing; has not been studied.

Signs and symptoms of hypersensitivity reaction (during infusion and for at least 4 hours after completion of infusion); lipid panel; anti-drug antibody assessment; hepatic function (ALT).

1 mg/kg IV every other week

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

Data not available

Safety and efficacy have not been established in patients younger than 1 month.

Consult WARNINGS section for additional precautions.