Generic drug: sebelipase alfa
Brand name: Kanuma
What is Kanuma (sebelipase alfa), and how does it work?
Kanuma (sebelipase alfa) injection 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.
What are the side effects of Kanuma?
Common side effects of Kanuma include:
- diarrhea,
- vomiting,
- nausea,
- fever,
- runny or stuffy nose,
- low levels of iron in the blood (anemia),
- cough,
- sore throat,
- hives,
- headache,
- weakness,
- constipation, and
- nausea.
What is the dosage for Kanuma?
Patients With Rapidly Progressive LAL Deficiency Presenting Within The First 6 Months Of Life
The recommended starting dosage is 1 mg/kg administered once weekly as an intravenous infusion. For patients who do not achieve an optimal clinical response, increase to 3 mg/kg once weekly.
Pediatric And Adult Patients With LAL Deficiency
The recommended dosage is 1 mg/kg administered once every other week as an intravenous infusion.
Preparation Instructions
Kanuma is for intravenous infusion only. Prepare Kanuma using the following steps.
Table 1: Total Infusion Volumes*
Weight Range (kg) | 1 mg/kg dose | 3 mg/kg dose† |
Total Infusion Volume (mL) | Total Infusion Volume (mL) | |
1 to 10.9 | 10 | 25 |
11 to 24.9 | 25 | 50 |
25 to 49.9 | 50 | 100 |
50 to 99.9 | 100 | 250 |
100 to 120.9 | 250 | 500 |
*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. †For patients with LAL deficiency presenting within the first 6 months of life who do not achieve an optimal clinical response with a dose of 1 mg/kg. |
- Determine the number of vials needed based on the patient's weight and the recommended dose of 1 mg/kg or 3 mg/kg, using the following calculations (a-b):
- Total dose (mg) = Patient's weight (kg) x 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
- 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 the 3 mg/kg dose or those who have experienced hypersensitivity reactions. 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.
Is Kanuma safe to use while pregnant or breastfeeding?
- There are no available data on Kanuma in pregnant women to inform any drug-associated risk.
- 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.
Latest Medications News
Daily Health News
Summary
Kanuma (sebelipase alfa) injection 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, a rare inherited disorder. Common side effects of Kanuma include diarrhea, vomiting, nausea, fever, runny or stuffy nose, low levels of iron in the blood (anemia), cough, sore throat, hives, headache, weakness, constipation, and nausea.
Multimedia: Slideshows, Images & Quizzes
-
Vitamin D Deficiency: How Much Vitamin D Is Enough?
What does vitamin D do? Learn about vitamin D benefits and discover foods that are high in vitamin D. Explore vitamin D...
-
Genetics: 11 Surprising Things Your Genes Say About You
Explore what role DNA plays in your health, love life, and more in this WebMD slideshow.
-
Magnesium: What Is it, Health Benefits, and Good Sources
Magnesium is a critical mineral nutrient that the body needs, along with calcium and potassium, to regulate blood pressure. Many...
-
Vitamin B6: Signs You're Not Getting Enough
B6 is a hard-working vitamin that affects everything from your mood to appetite to skin condition. Here are signs you may not...
-
Vitamins and Supplements: Signs You're Low on Vitamin B12
Your body needs vitamin B12 to keep blood cells healthy and to make your nerves work right. But what happens when you run low?
-
Family Health History: Genetics, DNA Testing and Your Health
WebMD explains why your doctor asks about your relatives' health conditions and how you can get the information if you don’t know.
Related Disease Conditions
-
Genetic Diseases
The definition of a genetic disease is a disorder or condition caused by abnormalities in a person's genome. Some types of genetic inheritance include single inheritance, including cystic fibrosis, sickle cell anemia, Marfan syndrome, and hemochromatosis. Other types of genetic diseases include multifactorial inheritance. Still other types of genetic diseases include chromosome abnormalities (for example, Turner syndrome, and Klinefelter syndrome), and mitochondrial inheritance (for example, epilepsy and dementia).
-
Vitamin D Deficiency
Vitamin D deficiency has been linked with rickets, cancer, cardiovascular disease, severe asthma in children and cognitive impairment in older adults. Causes include not ingesting enough of the vitamin over time, having limited exposure to sunlight, having dark skin, and obesity. Symptoms include bone pain and muscle weakness. Treatment for vitamin D deficiency involves obtaining more vitamin D through supplements, diet, or exposure to sunlight.
-
How Do I Know If I Have Telogen Effluvium or Androgenetic Alopecia?
What is the difference between telogen effluvium and androgenetic alopecia? Learn about these hair loss conditions and how to treat them.
-
Vitamin D Deficiency
-
What Foods to Avoid If You Have G6PD Deficiency
Glucose-6-phosphate dehydrogenase (G6PD) deficiency (G6PDD) is a genetic (hereditary) disorder. Therefore, you cannot prevent it. However, you can prevent the more serious G6PDD symptoms by avoiding certain foods such as fava beans, aniline dyes and certain medications.
-
Iron Deficiency
Iron is a mineral our bodies need. Iron deficiency is a condition resulting from not enough iron in the body. It is the most common nutritional deficiency and the leading cause in the US. Iron deficiency is caused due to increased iron deficiency from diseases, nutritional deficiency, or blood loss and the body's inability to intake or absorb iron. Children, teen girls, pregnant women, and babies are at most risk for developing iron deficiency. Symptoms of iron deficiency include feeling weak and tired, decreased work or school performance, slow social development, difficulty maintaining body temperature, decreased immune function, and an inflamed tongue. Blood tests can confirm an iron deficiency in an individual. Treatment depends on the cause of the deficiency. Proper diet that includes recommended daily allowances of iron may prevent some cases of iron deficiency.
-
What Are the 3 Stages of Iron Deficiency?
Iron is an important substance that our body needs in minute amounts. Daily ingestion of iron is needed for the health of red blood cells and muscle proteins, as well as the functioning of body cells. The normal body iron content in an adult is approximately 3-4 grams.
-
Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency (AATD) is an inherited disorder caused by mutations in the SERPINA1 gene. People with the condition are at risk for developing serious lung and liver disease.
-
What Are the Symptoms and Causes of Vitamin D Deficiency?
What is vitamin D deficiency? Learn the signs of vitamin D deficiency and what foods you can eat to help prevent vitamin D deficiency.
-
G6PD Deficiency
G6PD deficiency (Glucose 6-phosphate dehydrogenase) leads to a condition called hemolytic anemia. Causes of G6PD deficiency is an abnormal gene located in the X-chromosome, therefore, it is more common in males. Hemolytic anemia caused by G6PD deficiency generally occurs after exposure to malaria medications, antiitching drugs, and fava beans. Pneumonia and other infections can also precipitate hemolytic anemia in individuals with G6PD deficiency. Treatment is generally discontinuing the drug or compound treating infection. Blood transfusions are necessary in some individuals.
-
Genetic Counseling
Your health care provider may refer you to a genetic professional. Universities and medical centers also often have affiliated genetic professionals, or can provide referrals to a genetic professional or genetics clinic. Genetic counseling provides patients and family members the tools to make the right choice in regard to test for a disease or condition.
-
What Triggers G6PD Deficiency?
G6PD deficiency is a genetic disorder that results in an inadequate amount of a very important enzyme (protein) called glucose-6-phosphate dehydrogenase (G6PD) in the blood. The enzyme is required for the protection of the red blood cells and the regulation of various biochemical reactions in the body. When G6PD is deficient, red blood cells break down prematurely; this process is called hemolysis.
-
Tetrahydrobiopterin Deficiency
Tetrahydrobiopterin deficiency is a rare genetic disorder that increases the levels of phenylalanine and several other substances in the blood, at birth. Symptoms of Tetrahydrobiopterin deficiency include movement disorders, difficulty swallowing, seizures, and more. Causes of tetrahydrobiopterin deficiency can be from mutations of several genes including GCH1, PCBD1, PTS, and QDPR. Tetrahydrobiopterin deficiency is an inherited condition in an autosomal resessive pattern.
-
Genetic Testing: Families With Breast Cancer
Breast cancer can be a killer and the decision to get tested to see if a patient is prone to the disease should be discussed with a doctor -- particularly if the woman has a history of breast cancer in her family. Genetic testing can only tell so much about breast cancer risk, however.
-
Who Should Get Genetic Counselling?
Genetic counseling is the process that helps you in determining hereditary or genetic (running in your family) problems that might affect you or your family. It is done by a trained professional.
Treatment & Diagnosis
Medications & Supplements
Prevention & Wellness
Health Solutions From Our Sponsors

Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.