Hydrolytic Lysosomal Cholesteryl Ester-specific EnzymePrescription OnlyPregnancy Cat. Not classifiedFDA Yes
Kanuma
SEBELIPASE ALFA
1 INDICATIONS AND USAGE KANUMA ® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. KANUMA ® is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. ( 1 )
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Brand names:KANUMA
Quick Reference
Medicine ClassHydrolytic Lysosomal Cholesteryl Ester-specific Enzyme
FDA ApprovedYes
PregnancyCategory Not classified
Quick Facts
- Active ingredient
- SEBELIPASE ALFA
- Medicine class
- Hydrolytic Lysosomal Cholesteryl Ester-specific Enzyme
- Brand names
- KANUMA
- Availability
- Prescription only (Rx)
- Pregnancy category
- Not classified
- Half-life
- Half-life
- FDA approved
- Yes
What Is Kanuma?
1 INDICATIONS AND USAGE KANUMA ® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. KANUMA ® is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. ( 1 )
Kanuma (SEBELIPASE ALFA) belongs to the Hydrolytic Lysosomal Cholesteryl Ester-specific Enzyme class of medications. It was first approved by the FDA in Yes. This medication requires a prescription from a licensed healthcare provider.
Key Safety Information
- •Always follow the dose recommended by your healthcare professional. Taking more than prescribed increases the risk of side effects including serious complications.
- •Seek emergency care if you experience signs of an allergic reaction — hives, swelling, difficulty breathing, or rash. Stop the medication and call your doctor immediately.
- •Some medications can increase the risk of cardiovascular events. Tell your doctor if you have high blood pressure, a history of heart attack or stroke, or any pre-existing health condition.
- •Avoid or limit alcohol while taking Kanuma. Do not drink alcohol without first discussing it with your healthcare provider as it may increase adverse effects.
- •Take Kanuma with food or a full glass of water if stomach upset occurs. Stop use and talk to your doctor if you experience persistent stomach pain or GI symptoms.
- •Tell your doctor about all medications, supplements, and vitamins you take. Some combinations significantly increase the risk of harm.
This is a summary only. Always read the full prescribing information and consult your healthcare provider for personalized medical advice.
Uses & Indications
Kanuma is prescribed for the following conditions. Some uses are FDA-approved indications; others may be evidence-based off-label uses. Consult your healthcare provider for personalized guidance.
1 INDICATIONS AND USAGE KANUMA ® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
KANUMA ® is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
Dosage Guide
The following are general dosing guidelines only. Your actual dose should be determined by your healthcare provider based on your condition, renal/hepatic function, and other medications.
AD
Adult Dosage
2 DOSAGE AND ADMINISTRATION Administration of KANUMA should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) Infants with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life: The recommended starting dosage is 1 mg/kg as an intravenous infusion once weekly. ( 2.2 ) For patients with a suboptimal clinical response, increase the dosage to 3 mg/kg once weekly. ( 2.2 ) For patients with continued suboptimal clinical response, further increase the dosage to 5 mg/kg once weekly. ( 2.2 ) Pediatric and Adult Patients with LAL Deficiency: The recommended dosage is 1 mg/kg as an intravenous infusion once every other week. ( 2.2 ) For patients with a suboptimal clinical response, increase the dosage to 3 mg/kg once every other week. ( 2.2 ) See Full Prescribing Information for complete Dosage and Administration Information. Administration Instructions Infuse over at least 2 hours. ( 2.4
Available Forms
Available Strengths
Side Effects
Common
- 6 ADVERSE REACTIONS The most common adverse reactions are: Infants with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life (≥30%): diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, and urticaria.
- ( 6.1 ) Pediatric and Adult Patients with LAL Deficiency (≥8%): headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, and nausea.
- ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alexion at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice.
- In clinical trials, a total of 106 patients received treatment with KANUMA.
- The data described below reflect exposure to KANUMA in 75 patients who received KANUMA at dosages up to 3 mg/kg once weekly in clinical trials: Nine patients (5 males, 4 females) who had growth failure or other evidence of rapidly progressive LAL deficiency presenting within the first 6 months of life received KANUMA for up to 165 weeks (median 60 weeks) at escalating doses ranging between 0.35 mg/kg and 5 mg/kg once weekly [see Clinical Studies (14.1) ] .
- 66 pediatric and adult patients with LAL deficiency aged 4 to 58 years (33 males, 33 females) received KANUMA 1 mg/kg every other week for up to 36 weeks.
- Table 2 summarizes the most common adverse reactions occurring in >30% of patients with rapidly progressive LAL deficiency presenting within the first 6 months of life receiving KANUMA in Study
Serious
- 5 WARNINGS AND PRECAUTIONS Hypersensitivity to Eggs or Egg Products: Consider the risks and benefits of treatment in patients with known systemic hypersensitivity reactions to eggs or egg products.
- ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies, including KANUMA.
- These reactions in KANUMA-treated patients were based on application of Sampson criteria to identify signs/symptoms consistent with anaphylaxis.
- In clinical trials, 3 (infants) of 106 (3%) patients treated with KANUMA experienced signs and symptoms consistent with anaphylaxis.
- These patients experienced reactions during infusion with signs and symptoms including chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, swelling of eyelids, rhinorrhea, severe respiratory distress, tachycardia, tachypnea, and urticaria.
Rare
Medicine Interactions
Always inform your healthcare provider and pharmacist about ALL medications you take, including prescriptions, OTC medicines, vitamins, and supplements.
MajorModerateMinor
Warnings & Contraindications
5 WARNINGS AND PRECAUTIONS Hypersensitivity to Eggs or Egg Products: Consider the risks and benefits of treatment in patients with known systemic hypersensitivity reactions to eggs or egg products. ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies,
How Kanuma Works
12.1 Mechanism of Action LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase (LAL) enzyme. The primary site of action of the LAL enzyme is the lysosome, where the enzyme normally causes the breakdown of lipid particles, including LDL-c and triglycerides. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of blood vessels. The resulting lipid accumulation in the liver may lead to increased liver fat content and progression of liver disease, including fibrosis and cirrhosis. Lipid accumulation in the in
Pharmacokinetics
Half-Life
Half-life
Pregnancy & Breastfeeding Safety
Not classified
Breastfeeding
Many medications pass into breast milk in varying amounts. Before using Kanumawhile breastfeeding, discuss the benefits and risks with your healthcare provider or pharmacist — they can weigh your dose, your infant's age, and available lactation safety data to find the safest option for you and your baby.
Storage & Handling
Store KANUMA refrigerated at 2°C to 8°C (36°F to 46°F) in original carton to protect from light. Do not shake or freeze the vials.
Frequently Asked Questions
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Kanuma side effects
Complete adverse effect profile including common, serious, and rare reactions
Sources & References
- • FDA Prescribing Information — Official labeling and package insert for Kanuma
- • U.S. National Library of Medicine — MedlinePlus drug information
- • American Hospital Formulary Service (AHFS) — Clinical pharmacology monograph
- • PubMed / MEDLINE — View clinical studies
Last reviewed by MedCentralHub Medical Review Board · MedCentralHub Editorial Policy
Medical Disclaimer
The information on this page is for educational purposes only and is not intended as medical advice, diagnosis, or treatment. Always consult your doctor, pharmacist, or qualified healthcare provider before starting, stopping, or changing any medication.