taliglucerase alfa

Action and Spectrum

Actions and Spectrum: 

• The mechanism of action of taliglucerase alfa involves the replacement of the missing or deficient beta-glucocerebrosidase enzyme in the body. This enzyme is responsible for breaking down a specific type of fat called glucocerebroside. In people with Gaucher disease, the deficiency of this enzyme leads to the accumulation of glucocerebroside in various organs and tissues, which can cause a range of symptoms, including anemia, bone pain, and organ enlargement. 
• By providing the missing enzyme, taliglucerase alfa helps to break down the accumulated glucocerebroside, reducing the symptoms of Gaucher disease. taliglucerase alfa is administered intravenously and is typically given every two weeks. 
• taliglucerase alfa has a broad spectrum of activity, as it is effective in treating all types of Gaucher disease, including type 1, the most common form of the disease. It effectively reduces the symptoms of Gaucher disease, including anemia, bone pain, and organ enlargement, and improves the overall quality of life. 

Drug Interaction

Adverse Reaction

Frequency defined 

>10% 

Treatment naïve adolescents and children 

Vomiting (≥10%) 

Treatment-naïve adults 

Arthralgia (13%) 

Headache (19%) 

Switching from imiglucerase adults and children 

Headache (13%) 

Arthralgia (13%)  

1-10% 

Treatment-naïve adults 

Nausea (9%) 

Abdominal pain (6%) 

Flushing (6%) 

Urticaria (6%) 

Fatigue (9%) 

Dizziness (9%) 

Pruritus (6%) 

Vomiting (6%)  

Switch from imiglucerase adults and children. 

Pain in extremities (10%)  

Post-marketing reports 

Diarrhea 

Anaphylaxis 

Fatigue 

Back pain 

Type III immune-mediated fixed drug eruption 

Vomiting 

Black Box Warning

Contraindication / Caution

Contraindications/caution: 

Contraindications: 

• Hypersensitivity: taliglucerase alfa should not be used in individuals with known hypersensitivity or allergic reactions to the medication or its components. 
• Infusion-related reactions: taliglucerase alfa can cause infusion-related reactions, such as allergic reactions, hypersensitivity reactions, and anaphylaxis. Therefore, it should be used cautiously in individuals with a history of these reactions. 
• Pregnancy and breastfeeding: There is limited data on using taliglucerase alfa during pregnancy and breastfeeding. Therefore, it should only be used during pregnancy or breastfeeding if the potential benefits outweigh the risks. 

Caution: 

• Allergic reactions: Like with any medication, there is a risk of an allergic reaction to taliglucerase alfa. Signs of an allergic reaction may include hives, difficulty breathing, swelling of the face or throat, and dizziness. If you experience any of these symptoms, seek immediate medical attention. 
• Infusion reactions: taliglucerase alfa is given as an intravenous infusion and injected into a vein over time. Some people may experience infusion reactions like fever, chills, nausea, vomiting, headache, or rash. These symptoms usually go away independently, but you should inform your healthcare provider if you experience them. 
• Increased risk of infections: taliglucerase alfa may lower your body’s ability to fight infections.  
• Increased risk of bleeding: taliglucerase alfa may increase your risk of bleeding, particularly if you have a bleeding disorder or are taking medications that also increase your risk of bleeding.  
• Pregnancy and breastfeeding: The safety of taliglucerase alfa during pregnancy or breastfeeding is not fully known. 

Pregnancy / Lactation

Pregnancy consideration: Insufficient data available 

Lactation: Excretion of the drug in human breast milk is unknown 

Pregnancy category: 

Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.   

Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women. 

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.    

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: There is no data available for the drug under this category 

Pharmacology

Pharmacology: 

taliglucerase alfa is given as an intravenous infusion over some time. It has a plasma half-life of about 5 to 8 minutes, which means it is rapidly cleared from the bloodstream. However, it can accumulate in the reticuloendothelial system’s cells, including the liver, spleen, and bone marrow, where it can help break down glucocerebroside. 

Pharmacodynamics: 

taliglucerase alfa is a protein-based medication that induces an immune response in some patients. This can lead to the development of antibodies that can reduce the effectiveness of the medication over time. 

Overall, taliglucerase alfa is a well-tolerated medication that effectively treats the symptoms of type 1 Gaucher disease. 

Pharmacokinetics: 

Absorption 

taliglucerase alfa is administered by intravenous infusion, injected directly into a vein. This allows for rapid and complete absorption of the medication into the bloodstream.  

Distribution 

taliglucerase alfa is distributed throughout the body via the bloodstream. It is taken up by cells, which can help break down fatty substances accumulated in the cells due to the lack of functional glucocerebrosidase.  

Metabolism 

taliglucerase alfa is a recombinant protein produced using biotechnology rather than being synthesized by the body’s cells. As a result, it is not metabolized in the same way as endogenous proteins.  

Elimination and Excretion 

taliglucerase alfa is eliminated from the body primarily via the kidneys, although a small amount may also be eliminated via the feces. The half-life of taliglucerase alfa is approximately 4 hours, which means it is cleared from the body relatively quickly. 

Adminstartion

Administration: 

taliglucerase alfa is administered by intravenous infusion, injected directly into a vein over time. The dosage and frequency of administration of taliglucerase alfa depend on the individual patient’s weight and the severity of the disease. 

The recommended starting dose of taliglucerase alfa is 60 units/kg of body weight, administered once every other week. This dose may be adjusted based on the patient’s response to treatment and any adverse effects experienced. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: taliglucerase alfa 

Why do we use taliglucerase alfa? 

• taliglucerase alfa is a medication used to treat Gaucher’s disease. This genetic disorder results in the accumulation of a fatty substance called glucocerebroside in specific organs, particularly the spleen, liver, and bone marrow. This accumulation can cause various symptoms, including anemia, fatigue, bone pain and fractures, enlarged spleen and liver, and an increased risk of infections. 
• taliglucerase alfa is an enzyme replacement therapy that replaces the deficient enzyme beta-glucocerebrosidase, which breaks down glucocerebroside. By providing the body with this missing enzyme, taliglucerase alfa can help to reduce the accumulation of glucocerebroside and improve symptoms of Gaucher disease.