Sebelipase Alfa

Sebelipase Alfa is a prescription medication used for enzyme replacement in patients with lysosomal acid lipase (LAL) deficiency.

• Sebelipase Alfa is available under the following different brand names: Kanuma.

Common side effects of Sebelipase Alfa include:

• Fever,
• Weakness,
• Runny nose,
• Sinus pain,
• Sore throat,
• Cough,
• Rash,
• Diarrhea,
• Constipation,
• Nausea,
• Vomiting, and
• Headache

Serious side effects of Sebelipase Alfa include:

• Hives,
• Difficulty breathing,
• Swelling of the face, lips, tongue, or throat,
• Itching,
• Flushed,
• Chills,
• Fever,
• Runny nose,
• Eye irritation,
• Diarrhea,
• Chest discomfort,
• Severe skin rash,
• Swelling,
• Agitation,
• Irritability,
• Stomach pain,
• Pale skin,
• Lightheadedness,
• Shortness of breath,
• Rapid heart rate, and
• Difficulty concentrating

Rare side effects of Sebelipase Alfa include:

• None 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Solution for injection

• 20 mg/10 mL per vial (2 mg/mL)

Lysosomal Acid Lipase Deficiency

Adult dosage

• 1 mg/kg intravenous infusion every other week
• Suboptimal clinical response: Increase to 3 mg/kg intravenous infusion every other week.

Pediatric dosage

• 1 mg/kg intravenous infusion every other week
• Suboptimal clinical response: Increase to 3 mg/kg intravenous infusion every other week.
• Rapidly progressive LAL deficiency presenting within the first 6 months of life.
• Starting dose: 1 mg/kg IV infusion once weekly
• Suboptimal clinical response: Increase to 3 mg/kg once weekly.
• Continued suboptimal clinical response on 3 mg/kg/week: Further increase to 5 mg/kg intravenous infusion once weekly.

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Sebelipase Alfa has no noted severe interactions with any other drugs.
• Sebelipase Alfa has no noted serious interactions with any other drugs.
• Sebelipase Alfa has no noted moderate interactions with any other drugs.
• Sebelipase Alfa has no noted minor interactions with any other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

1. None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Sebelipase Alfa?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Sebelipase Alfa?”

Cautions

• Hypersensitivity reactions
  • Hypersensitivity, including anaphylaxis, was reported as early as the sixth infusion and as late as 1 year after treatment initiation.
  • Owing to the potential for anaphylaxis, assure appropriate medical support is readily available during the administration.
  • Management based on reaction severity and may include temporarily interrupting infusion, lowering infusion rate, and/or treatment with antihistamines, antipyretics, and/or corticosteroids.
  • If interrupted, may resume infusion at a slower rate with increases as tolerated.
  • Pretreatment with antipyretics and/or antihistamines may prevent subsequent reactions in those cases where symptomatic treatment was required.
  • If a severe hypersensitivity reaction occurs, immediately discontinue the infusion, and initiate appropriate medical treatment.
• Hypersensitivity to eggs or egg products
  • Product is produced in the egg whites of genetically engineered chickens.
  • Patients with a known history of egg allergies were excluded from clinical trials.
  • Consider risks and benefits with known systemic hypersensitivity reactions to eggs or egg products.

Pregnancy and Lactation

• There are no available data on pregnant women to inform of any drug-associated risk.
• Animal reproductive studies conducted with Sebelipase Alfa showed no evidence of embryo lethality, fetotoxicity, teratogenicity, or abnormal early embryonic development at dosages up to 164 and 526 times the human dosage of 1 mg/kg every other week (based on AUC) in rats and rabbits, respectively.
• Lactation
  • Unknown if distributed in human breast milk.