Sebelipase alfa

What is Sebelipase alfa?

• Sebelipase alfa (KANUMA) is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme used to treat patients with a rare disease known as lysosomal acid lipase (LAL) deficiency.

What are the uses of this medicine?

• This medicine is used for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.

How does this medicine work?

• Sebelipase (se" be lye' pase) alfa is a recombinant form of the lysosomal enzyme that is absent or deficient in inherited conditions that are marked by dyslipidemia, early onset of atherosclerosis and fatty liver disease that can progress to cirrhosis.
• The severe form of lysosomal acid lipase deficiency is known as Wolman disease in which the enzyme is entirely absent.
• Wolman disease is a severe, progressive disease of infancy marked by severe diarrhea, failure to thrive and progressive hepatic fibrosis and cirrhosis, usually leading to death within the first year of life.
• The same enzyme defect also appears to be responsible for the milder form of dyslipidemia and liver disease known as cholesterol ester storage disease in which the enzyme is deficient, but not totally absent.
• Cholesterol ester storage disease typically presents in adolescence or young adulthood, but may go undetected and present with middle age with wide spread complications of atherosclerosis or cirrhosis of unknown cause.
• The enzyme acts in lysosomes upon cholesterol esters and its deficiency results in accumulation of the cholesterol esters and triglycerides in hepatocytes and diffusely in macrophages in the liver, spleen, adrenals, bone marrow, lymph nodes and intestinal villi. Liver histology demonstrates microvesicular steatosis, foamy macrophages and progressive fibrosis.
• Serum aminotransferase levels are typically mildly or moderately elevated.
• Cholesterol lowering agents have been used in an attempt to treat these conditions, but have little effect.
• Cirrhosis and end stage liver disease can be managed successfully with liver transplantation, and hematopoietic cell transplantation has been reported to reverse some of the manifestations.
• Recently, infusions of recombinant lysosomal acid lipase have been found to decrease serum enzyme elevations and result in resolution of hepatomegaly in patients with cholesterol ester storage disease.
• In some cases, the beneficial effects were dramatic with evidence of disease regression. Pilot studies in children with Wolman disease have also had promising results with enzyme replacement.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No clinically important drug interactions have been observed with KANUMA.

Is this medicine FDA approved?

• It was approved for use in the United States in 2015.

How should this medicine be used?

Recommended Dosage: 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.

Administration

• KANUMA is for intravenous infusion only.
• Infuse over at least 2 hours.
• Consider further prolonging the infusion time for the 3 mg/kg dose or if a hypersensitivity reaction occurs.
• Consider a 1-hour infusion for the 1 mg/kg dose in patients who tolerate the infusion.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Injection: 20 mg/10 mL (2 mg/mL) solution in single-use vials

This medicine is available in fallowing brand namesː

• KANUMA

What side effects can this medication cause?

The most common side effects of this medicine include:

Patients with Rapidly Progressive Disease Presenting within the First 6 Months of Life

• diarrhea
• vomiting
• fever
• rhinitis
• anemia
• cough
• nasopharyngitis
• urticaria

Pediatric and Adult Patients

• headache
• fever
• oropharyngeal pain
• nasopharyngitis
• asthenia
• constipation
• nausea

What special precautions should I follow?

• Hypersensitivity reactions, including anaphylaxis, have been reported in KANUMA. Observe patients during and after the infusion. Consider interrupting the infusion or lowering the infusion rate, based on the severity of the reaction. If a severe hypersensitivity reaction occurs, immediately stop the infusion and initiate appropriate treatment. Pre-treatment with antipyretics and/or antihistamines may prevent subsequent reactions in those cases where symptomatic treatment is required.
• KANUMA is produced in the egg whites of genetically engineered chickens. Patients with a known history of egg allergies were excluded from the clinical trials. Consider the risks and benefits of treatment in patients with known systemic hypersensitivity reactions to eggs or egg products.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no available data on KANUMA in pregnant women to inform any drug-associated risk.

Can this medicine be used in children?

• Safety and effectiveness of KANUMA have been established in pediatric patients aged 1 month and older.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• Sebelipase alfa

Inactive ingredients:

• Trisodium citrate dihydrate
• Citric acid monohydrate
• Albumin Human

Who manufactures and distributes this medicine?

Manufactured by: Alexion Pharmaceuticals Inc. 121 Seaport Boulevard, Boston KANUMA is a registered trademark of Alexion Pharmaceuticals Inc.

What should I know about storage and disposal of this medication?

• Store KANUMA under refrigeration between 2°C to 8°C (36°F to 46°F) in original carton to protect from light.
• Do not shake or freeze the vials.

• Dailymed label info on Sebelipase alfa
• FDA Sebelipase alfa

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