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Sebelipase Alfa - Dosage, Indications, and Side Effects

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Sebelipase alfa is a drug that is used in the treatment of lysosomal acid lipase deficiency. The following article provides particulars on Sebelipase alfa.

Written by

Dr. Sri Ramya M

Medically reviewed by

Dr. Kaushal Bhavsar

Published At March 30, 2023
Reviewed AtMarch 30, 2023

Overview

A Sebelipase alfa is a recombinant form of the lysosomal acid lipase enzyme. It is used in the treatment of lysosomal acid lipase deficiency disease. Lysosomal acid lipase is naturally produced in the body. It helps in the breakdown of fats and prevents the buildup of fat in the cells. Lysosomal acid lipase deficiency is a dreadful genetic disorder that damages the organs of the body and results in early death.

Sebelipase alfa was developed by Synageva and was approved for use in 2015 in the European Union and the United States. It was approved by the FDA (the food and drug administration) in 2015. The recombinant form of lysosomal acid lipase is produced from chickens that are genetically modified. The enzyme is purified after extraction, and it becomes available for use.

How Does Sebelipase Alfa Work?

Lysosomal acid lipase deficiency is a genetic condition in which this enzyme is absent, and this causes problems in the breakdown and use of fats and cholesterol that are present in the body. This leads to the accumulation of harmful amounts of fats, resulting in liver diseases and other organ failures. A Sebelipase alfa is a recombinant form of this enzyme. It binds to the receptors on the cell surface and enters the lysosomes in the cells. It breaks down the cholesteryl esters and triglycerides to free cholesterol, free fatty acids, and glycerol in the lysosomes, thereby preventing the accumulation of harmful fats and cholesterol.

What Are the Uses of Sebelipase Alfa?

Sebelipase alfa is used in the treatment of lysosomal acid lipase deficiency. It is used as a long-term enzyme replacement therapy in individuals with lysosomal acid lipase deficiency.

What Is the Dosage of Sebelipase Alfa?

Sebelipase alfa is available as an intravenous solution. It is available as a 2 mg/mL solution in 20 mg/10 mL vials. The recommended adult dose for lysosomal acid lipase deficiency is 1 mg (milligram) per kg (kilogram) of Sebelipase alfa over an intravenous infusion every other week. The dose is increased to 3 mg/kg every other week for patients with suboptimal clinical response.

  • The recommended pediatric dose for rapidly progressing lysosomal acid lipase deficiency for patients within the first six months of life is 1mg/kg over an intravenous infusion once a week. The dose is increased to 3 mg/kg once a week for patients with suboptimal clinical response, and it is increased to 5 mg/kg for patients with continued suboptimal clinical response.

  • The recommended dose for pediatric patients over six months of age is 1 mg/kg over an intravenous infusion every other week. The dose is increased to 3 mg/kg for patients with suboptimal clinical response.

Warnings:

1. All the instructions on the medicine label and package should be followed.

2. Sebelipase alfa should not be used if an allergy to egg or egg products is present.

3. Sebelipase alfa may harm the unborn baby and should not be used if a person is pregnant or planning to become pregnant.

4. Sebelipase alfa is not recommended for breastfeeding mothers.

5. It is not approved for use in pediatric patients younger than one month.

6. It is not recommended for patients above 65 years of age.

For Patients

What Is Lysosomal Acid Lipase Deficiency?

Lysosomal acid lipase deficiency is a chronic, inherited disorder that affects the body’s ability to produce lysosomal acid lipase. Lysosomal acid lipase is essential for the breakdown of fats and cholesterol, including low-density lipoproteins (LDL). This enzyme deficiency results in the accumulation of fats in the organs and tissues throughout the body, including the liver, spleen, and blood vessel walls, resulting in liver diseases and other organ diseases. High levels of LDL cholesterol are associated with complications like heart attack, stroke, liver fibrosis, liver cirrhosis, and liver failure.

What Causes Lysosomal Acid Lipase Deficiency?

Lysosomal acid lipase deficiency is an inherited, inborn error of metabolism that causes lysosomal storage disease. This deficiency is due to a mutation in the LIPA (lipase A, lysosomal acid type) gene that is responsible for the coding of the lysosomal lipase protein. This results in the accumulation of fats in the organs and tissues throughout the body, including the liver, spleen, and blood vessel walls, resulting in liver diseases and other organ diseases.

What Happens in Lysosomal Acid Lipase Deficiency?

Lysosomal lipase acid deficiency causes frequent vomiting, diarrhea, weight loss, swelling of the abdomen, and poor weight gain in infants. As the disease progresses, fat accumulation in the liver results in complications like yellowing of the skin and the eyes and a persistent low-grade fever. It gradually results in life-threatening complications like low levels of circulating red blood cells, liver dysfunction, and physical wasting. Adults generally present with headache, fever, oropharyngeal pain, nasopharyngitis, nausea, constipation, asthenia, diarrhea, stomach pain, malabsorption, and vomiting. It also causes bile duct problems and liver diseases. Pediatric patients with rapidly progressing lysosomal acid lipase deficiency present with fever, cough, anemia, diarrhea, vomiting, rhinitis, urticaria, and nasopharyngitis.

Learn More About Sebelipase Alfa

Facts to Know Before Starting Sebelipase Alfa:

Why and When to Switch to Sebelipase Alfa?

There is no cure for lysosomal acid lipase deficiency. The only treatment option available is enzyme replacement therapy with the recombinant form of this enzyme. Sebelipase alfa is the only FDA-approved drug for the treatment of lysosomal acid lipase deficiency. This drug is administered over an intravenous infusion every two weeks for adults and children.

How Effective Is Sebelipase Alfa?

Sebelipase alfa is very effective in treating this disorder. It showed significant improvement in parameters such as liver transaminases, hepatomegaly, and dyslipidemia in children and adults with lysosomal acid lipase deficiency.

Things to Inform the Doctor Before Sebelipase Alfa Is Prescribed:

1. The presence of drug allergies or allergies to eggs or egg products should be informed.

2. The presence of anemia or other medical conditions should be informed.

3. Sebelipase alfa may harm the unborn baby and should not be consumed if a person is pregnant or planning to become pregnant. Pregnancy or pregnancy planning should be discussed with the doctor before starting this drug.

4. It is not recommended for breastfeeding mothers, and they should be informed before starting this drug.

Starting Sebelipase Alfa:

Sebelipase alfa is given intravenously (into a vein) by a healthcare provider. Sebelipase alfa is not available for oral use. It is usually given once every other week. It is given slowly over one or two hours. The patient is monitored for allergic reactions during the administration of Sebelipase alfa.

Things to Do While Under Sebelipase Alfa

1. It is important to follow the instructions given by the doctor while taking Sebelipase alfa.

2. The presence of any allergic reactions or abnormal changes should be informed to the doctor.

3. Any weight changes should be informed.

4. It is important to inform the doctor of missing an appointment.

5. The doctor’s instructions about food or drink restrictions and restrictions on activities should be followed.

Advice for Caregivers:

The patient’s caregivers should inform the doctor if signs of allergic reactions like hives, breathing difficulties, and swelling of the face, lips, and throat are present.

Things to Do After Taking Sebelipse Alfa:

It is important to inform the doctor if any noticeable changes are present after taking Sebelipase alfa. Any signs of allergic reactions like difficulty breathing, hives, swelling of the face, lips, or tongue, or other symptoms like stomach pain, agitation, fever, weakness, rapid weight loss, nausea, vomiting, and diarrhea should be informed immediately.

Diet Modifications:

Diet modifications are not necessary unless specified by the doctor. A regular diet that is clean, healthy, and balanced can be followed. However, alcohol intake and smoking should be avoided.

Look Out for the Side Effects:

On experiencing side effects, it is essential to inform the doctor to adjust the treatment accordingly. The patients should familiarize themselves with the common and rare side effects of Sebelipase alfa so that patients know what to look out for. Sebelipase alfa may cause some other side effects that are not listed here. The common side effects include:

  • Abdomen pain.

  • Fever.

  • Weakness.

  • Chills.

  • Coughing.

  • Diarrhea.

  • Anxiety.

  • Blurred vision.

  • Dry mouth.

  • Dizziness.

  • Headache.

  • Nervousness.

  • Skin rashes.

  • Nausea and vomiting.

The less common side effects include:

  • Chest discomfort.

  • Irregular heartbeat.

  • Shallow breathing.

  • Redness of the skin.

  • Swelling of the eyelids.

Staying on Sebelipase Alfa:

Tips to Stay on Track:

The improvement in the symptoms of this disease after taking Sebelipase alfa does not happen overnight. Sticking to a proper dosage schedule is essential, and the dosage should not be missed. If any side effects occur after taking this drug, it is essential to seek medical help. Skipping appointments or stopping the medication should not be done without the doctor’s advice. It should be confirmed with the physician if an appointment is missed.

Things to remember,

1) Sticking to the dosage schedule is essential because missing a dose will affect the outcome of the treatment.

2) It is essential to follow up regularly with the doctor and inform them about improvements in the symptoms.

For Doctors

Indications:

Sebelipase alfa is indicated in the treatment of lysosomal acid lipase deficiency. It is used as a long-term enzyme replacement therapy in individuals with lysosomal acid lipase deficiency.

Mechanism of Action:

Lysosomal acid lipase deficiency is a genetic condition in which this enzyme is absent, and this causes problems in the breakdown and use of fats and cholesterol that are present in the body. This leads to the accumulation of harmful amounts of fats, resulting in liver diseases and other organ failures. A sebelipase alfa is a recombinant form of this enzyme. It binds to the receptors on the cell surface and enters the lysosomes in the cells. It breaks down the cholesteryl esters and triglycerides to free cholesterol, free fatty acids, and glycerol in the lysosomes, thereby preventing the accumulation of harmful fats and cholesterol.

Pharmacodynamics:

Sebelipas alfa acts as a replacement enzyme for patients with lysosomal acid lipase deficiency caused by a genetic disorder. Sebelipase alfa is produced from the eggs of genetically engineered chickens through recombinant DNA (deoxyribonucleic acid) technology. Sebelipase alfa binds to the surface receptors on the cells through glycans expressed on the proteins and enters the lysosomes. It catalyzes the lysosomal hydrolysis of the cholesteryl esters and triglycerides to free cholesterol, free fatty acids, and glycerol.

Pharmacokinetics:

Absorption:

Nonlinear pharmacokinetics is greater than the dose-proportional increase in exposure that is observed at 1 to 3 mg/kg dose. Substantial accumulation is not present following repeated dosing. Peak plasma concentrations are observed at 1.1 to 1.3 hours after a single dose in pediatric and adult patients. Increased LDL-cholesterol and triglyceride levels decreased within eight weeks of treatment. Decreased ALT (alanine transaminase) concentrations were observed within two weeks of treatment with Sebelipase alfa.

Distribution:

The distribution of Sebelipase alfa in breast milk is not known.

Metabolism:

Sebelipase alfa is metabolized through catabolism. It is catabolized into smaller peptides and amino acids.

Elimination:

The mean half-life of Sebelipase alfa is approximately six minutes following intravenous administration.

What Are the Ingredients in Sebelipase Alfa?

Each mL of solution contains 2 mg of Sebelipase alfa, 1.57 mg of citric acid monohydrate, 10 mg of human serum albumin, and 13.7 mg of trisodium citrate dihydrate.

Warnings and Precautions:

1. Hypersensitivity Reactions: Hypersensitivity reactions may occur within four hours of administration. The patients should be monitored with appropriate resuscitation measures.

2. Egg Allergy: Sebelipase alfa may cause allergic reactions if a patient is allergic to egg or egg products.

3. Antibody Formation: The development of antibodies to Sebelipase alfa, such as neutralizing, has been reported.

4. Pregnancy: No information is available regarding adverse embryo-fetal effects. However, Sebelipase alfa is not recommended for pregnant women.

5. Lactation: Not known whether it is distributed into breast milk. However, Sebelipase alfa is not recommended for lactating mothers.

Indications and Uses:

Sebelipase alfa is used in the treatment of lysosomal acid lipase deficiency. It is used as a long-term enzyme replacement therapy in individuals with lysosomal acid lipase deficiency.

Dosage:

Sebelipase alfa is available as an intravenous solution. It is available as a 2 mg/mL solution in 20 mg/10 mL vials.

  • The recommended adult dose for lysosomal acid lipase deficiency is 1 mg (milligram) per kg (kilogram) of Sebelipase alfa over an intravenous infusion every other week. The dose is increased to 3 mg/kg every other week for patients with suboptimal clinical response.

  • The recommended pediatric dose for rapidly progressing lysosomal acid lipase deficiency for patients within the first six months of life is 1mg/kg over an intravenous infusion once a week. The dose is increased to 3 mg/kg once a week for patients with suboptimal clinical response, and it is increased to 5 mg/kg for patients with continued suboptimal clinical response.

  • The recommended dose for pediatric patients over six months of age is 1 mg/kg over an intravenous infusion every other week. The dose is increased to 3 mg/kg for patients with suboptimal clinical response.

Considerations for Administration:

  • Sebelipase alfa is administered through intravenous infusion. It is infused for one or two hours. Longer infusion is considered for patients receiving a 3 mg/kg dose.

  • Sebelipase alfa should not be given if an allergy to egg or egg products is present.

  • Sebelipase alfa may harm the unborn baby and should not be used if a person is pregnant or planning to become pregnant.

  • It is not recommended for breastfeeding mothers.

  • It is not approved for use in pediatric patients younger than one month.

Contraindications:

Sebelipase alfa should not be prescribed for patients with known hypersensitivity to Sebelipase alfa or its inactive ingredients.

Results from Clinical Trials:

A 20-week, double-blind, placebo-controlled study was conducted to demonstrate the safety and efficacy of Sebelipase alfa. The results from the phase III study showed early and rapid improvements in liver injury markers and lipid abnormalities with Sebelipase alfa treatment, and no progression of liver disease was noted for up to five years.

Drug Interactions:

Formal drug interaction studies for Sebelipase alfa have not been performed.

Common Adverse Effects:

The common adverse effects reported are runny nose, sore throat, sinus pain, cough, rashes, diarrhea, nausea, vomiting, and constipation.

Other Specifications:

  • Hypersensitivity Reactions: Hypersensitivity reactions may occur within four hours of administration. The patients should be monitored with appropriate resuscitation measures.

  • Egg Allergy: Sebelipase alfa may cause allergic reactions if a patient is allergic to egg or egg products.

  • Antibody Formation: The development of antibodies to Sebelipase alfa, such as neutralizing, has been reported.

  • Sebelipase Alfa During Pregnancy: No studies have been conducted on using Sebelipase alfa in pregnant women. Therefore, there is no information regarding the risk of birth defects and miscarriage due to Sebelipase alfa use in pregnant women.

  • Sebelipase Alfa During Lactation: No information is available on the presence of Sebelipase alfa in human milk. However, Sebelipase alfa should not be used in nursing mothers.

  • Pediatric Use: The safety of Sebelipase alfa use in pediatric patients less than one month of age has not been demonstrated. Hence, its use is not recommended in such patients.

  • Geriatric Use: The safety and efficacy of Sebelipase alfa in patients above 65 years of age have not been established. Hence, Sebelipase alfa is not recommended in such patients.

  • Hepatic Impairment: Sebelipase alfa should not be used in patients with moderate or severe liver disease.

  • Renal impairment: No information is available regarding dose adjustments for patients with renal diseases.

  • Hepatic Impairment: No information is available regarding dose adjustments for patients with liver diseases.

Source Article IclonSourcesSource Article Arrow
Dr. Kaushal Bhavsar
Dr. Kaushal Bhavsar

Pulmonology (Asthma Doctors)

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