Dosage & Administration
Administration of KANUMA should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis.
Infants with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life:
Pediatric and Adult Patients with LAL Deficiency:
See Full Prescribing Information for complete Dosage and Administration Information.
Administration Instructions
By using PrescriberAI, you agree to the AI Terms of Use.
Kanuma Prescribing Information
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate KANUMA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue KANUMA and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1)].
KANUMA® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
Recommendations Prior to KANUMA Treatment
Administration of KANUMA should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1)].
Initiate KANUMA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1)].
Recommended Dosage
Infants with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life:
- The recommended starting dosage is 1 mg/kg administered as an intravenous infusion once weekly.
- For patients with a suboptimal clinical response, increase the dosage to 3 mg/kg once weekly.
- For patients with continued suboptimal clinical response on the 3 mg/kg once weekly dosage, further increase the dosage to 5 mg/kg once weekly.
- A suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly.
Pediatric and Adult Patients with LAL Deficiency:
- The recommended dosage is 1 mg/kg administered as an intravenous infusion once every other week.
- For patients with a suboptimal clinical response, increase the dosage to 3 mg/kg once every other week.
- A suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers [e.g., alanine aminotransferase (ALT), aspartate aminotransferase (AST)], and/or parameters of lipid metabolism [e.g., low-density lipoprotein cholesterol (LDL-c), triglycerides (TG)].
Preparation Instructions
KANUMA is for intravenous infusion only. Prepare KANUMA using the following steps.
- Determine the number of vials needed based on the patient's weight and the recommended dose of 1 mg/kg, 3 mg/kg, or 5 mg/kg using the following calculations (a-b):
- Total dose (mg) = Patient's weight (kg) × Recommended dose (mg/kg)
- Total number of vials = Total dose (mg) divided by 20 mg/vial
- Round to the next whole vial and remove the required number of vials from the refrigerator to allow them to reach room temperature.
- Volume (mL) of calculated total dose = Total dose (mg) divided by the 2 mg/mL concentration
- Volume (mL) of 0.9% Sodium Chloride for dilution = Total infusion volume (mL) for patient's weight (see Table 1) - Volume (mL) of calculated total dose
Table 1: Total Infusion Volumes * Weight Range (kg) 1 mg/kg dose 3 mg/kg dose 5 mg/kg dose Total Infusion Volume (mL) Total Infusion Volume (mL) Total Infusion Volume (mL) - *
- The infusion volume should be based on the prescribed dose and should be prepared to a final KANUMA concentration of 0.1 mg/mL to 1.5 mg/mL.
1 to 2.9 4 8 12 3 to 5.9 6 12 20 6 to 10.9 10 25 50 11 to 24.9 25 50 150 25 to 49.9 50 100 250 50 to 99.9 100 250 500 100 to 120.9 250 500 600 - Mix gently by inversion. Do not shake the vials or the prepared infusion.
- The solution should be inspected visually for particulate matter and discoloration prior to administration. The solution should be a clear to slightly opalescent, colorless to slightly colored solution. Thin, translucent particles or fibers may be present in the vials or diluted solution. Do not use if the solution is cloudy or if other particulate matter is observed.
- Vials are for single-use only. Discard any unused product. Do not freeze.
Administration Instructions
Administer the diluted solution as an intravenous infusion using a low-protein binding infusion set with an in-line, low-protein binding 0.2 micron filter.
Infuse over at least 2 hours. Consider further prolonging the infusion time for patients receiving dosages greater than 1 mg/kg or those who have experienced hypersensitivity reactions [see Warnings and Precautions (5.1)]. A 1-hour infusion may be considered for those patients receiving the 1 mg/kg dose who tolerate the infusion.
Storage of Diluted Solution
KANUMA contains no preservatives; therefore, product should be used immediately after dilution. If immediate use is not possible, the diluted product may be stored up to 24 hours in the refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze or shake. Protect from light.
Injection: 20 mg/10 mL (2 mg/mL) clear to slightly opalescent, colorless to slightly colored solution in single-dose vials.
Pregnancy
Risk Summary
Available data with KANUMA use in pregnant women are insufficient to identify a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. Animal reproductive studies conducted with sebelipase alfa showed no evidence of embryolethality, 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.
The background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.
Animal Data
Sebelipase alfa administered during the period of organogenesis to rats (on gestation days 6, 9, 12, 15 and 17) and rabbits (on gestation days 7, 10, 13, 16 and 19) at intravenous doses up to 60 and 50 mg/kg, respectively, (approximately 164 and 526 times the human AUC of 1387 ng.h/mL at 1 mg/kg dose administered once every other week, respectively) did not cause any adverse effects on embryofetal development. A pre- and post-natal development study in rats showed no evidence of adverse effects on pre- and postnatal development at intravenous doses (administered on gestation days 6, 9, 12, 15, 18, and 20 and days 4, 7, 10, 14, and 17 postpartum) of sebelipase alfa up to 60 mg/kg/day (approximately 164 times the human AUC of 1387 ng.h/mL at 1 mg/kg dose administered once every other week).
Lactation
Risk Summary
There are no data on the presence of sebelipase alfa in human milk, the effects on the breastfed infant, or the effects on milk production. It is not known if sebelipase alfa is present in animal milk. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for KANUMA and any potential adverse effects on the breastfed infant from sebelipase alfa or from the underlying maternal condition.
Pediatric Use
Safety and effectiveness of KANUMA have been established in pediatric patients aged 1 month and older. Clinical trials with KANUMA were conducted in 56 pediatric patients (range 1 month to <18 years old) [see Clinical Studies (14)].
Geriatric Use
Clinical trials of KANUMA did not include any patients aged 65 years old and older. It is not known whether they respond differently than younger patients.
None.