Taliglucerase alfa is a medication used primarily for the treatment of Gaucher disease, which is a rare genetic disorder. People with Gaucher disease lack sufficient activity of an enzyme called glucocerebrosidase, which is necessary for the breakdown of glucocerebroside, a type of fat. As a result, glucocerebroside accumulates in various body tissues, leading to symptoms such as enlargement of the liver and spleen, bone abnormalities, and blood disorders.

Taliglucerase alfa serves as an enzyme replacement therapy (ERT). It provides patients with a recombinant form of human glucocerebrosidase, which helps to reduce the buildup of glucocerebroside in the body's cells. This drug is particularly notable because it is the first plant cell-based ERT for Gaucher disease, produced in modified carrot cells, which differentiates it from other ERTs that are typically produced in mammalian cell systems.

Glucocerebrosidase (Taliglucerase alfa Injection) is a plant-derived recombinant enzyme replacement therapy that has been approved in children and adults with Type-1 Gaucher disease.

Glucocerebrosidase (Taliglucerase alfa Injection) Uses:

• Gaucher disease:
  • It is used in the management of adult and pediatric patients with a confirmed diagnosis of type 1 Gaucher disease.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in Adults

Taliglucerase alfa is a medication used to treat Gaucher disease, a condition where a specific type of fat builds up in the body because it lacks a necessary enzyme. This medicine, made from modified carrot cells, is given through an IV and helps reduce this fat buildup. Although it's generally safe, it can sometimes cause reactions during infusion, such as allergies.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in the treatment of Gaucher disease (type 1):

• For treating Gaucher disease type 1, taliglucerase alfa is typically given as an IV injection at a dose of 60 units per kilogram of body weight every two weeks, though the exact dose can vary depending on how severe the disease is.

Conversion from imiglucerase:

• If a patient is switching from another treatment called imiglucerase, they can start taliglucerase alfa at the same dose they were taking of imiglucerase, also every two weeks.

Note: This approach of switching treatments is based on the results from a study that included patients who had been stable on imiglucerase for at least six months.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in Children

Taliglucerase alfa is a medication used to treat Gaucher disease, a condition where a specific type of fat builds up in the body because it lacks a necessary enzyme. This medicine, made from modified carrot cells, is given through an IV and helps reduce this fat buildup. Although it's generally safe, it can sometimes cause reactions during infusion, such as allergies.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in the treatment of Gaucher disease (type 1):

For children aged 4 years and older, and for adolescents with Gaucher disease type 1:

• The recommended dose of taliglucerase alfa is 60 units per kilogram, given through IV every two weeks.
• Conversion from imiglucerase:
  • If a patient is switching from another enzyme replacement therapy called imiglucerase, they should start taliglucerase alfa at the same dose they were previously taking, also administered every two weeks.
  • The dose can be adjusted if needed to better manage the disease.

Note: The guidance for switching from imiglucerase to taliglucerase alfa comes from a study that focused on patients who had been stable on imiglucerase for at least six months.

Taliglucerase alfa (Elelyso) Pregnancy Category: B

• In studies on animals, taliglucerase alfa did not show any harmful effects on reproduction.
• However, pregnancy can worsen the symptoms of type I Gaucher disease or cause new symptoms to appear in women with the condition.
• If the disease is not well managed during pregnancy, there is a higher risk of miscarriage.
• Additionally, adverse outcomes like enlargement of the liver and spleen and low platelet count, which can lead to increased bleeding and the need for blood transfusions after giving birth, may occur.

Use of Taliglucerase alfa during lactation:

• It's unclear whether taliglucerase alfa passes into breast milk.
• Even if it does, any enzyme ingested by a nursing infant would likely be broken down by their digestive system and not have an effect.
• However, breastfeeding could potentially lead to further bone loss in mothers with Gaucher disease.
• The manufacturer advises that the decision to continue or stop breastfeeding while on this medication should consider both the potential risks to the infant and the health benefits for the mother.
• This balance is important to ensure the well-being of both mother and child.

Taliglucerase alfa Dose in Kidney Disease:

• The manufacturer's guidelines for taliglucerase alfa do not provide any specific dosage adjustments for patients with kidney disease.

Taliglucerase alfa Dose in Liver disease:

• The manufacturer's labeling for taliglucerase alfa does not specify any required dosage adjustments for patients with liver disease.

Common Side Effects of Glucocerebrosidase (Taliglucerase alfa Injection):

• Central Nervous System:
  • Headache
• Gastrointestinal:
  • Vomiting
• Hypersensitivity:
  • Hypersensitivity Reaction
• Immunologic:
  • Antibody Formation
• Neuromuscular & Skeletal:
  • Arthralgia
  • Limb Pain
• Respiratory:
  • Pharyngitis

Less Common Side Effects of Glucocerebrosidase (Taliglucerase alfa Injection):

• Cardiovascular:
  • Flushing
• Central Nervous System:
  • Dizziness
  • Fatigue
• Dermatologic:
  • Pruritus
  • Urticaria
  • Skin Rash
• Gastrointestinal:
  • Nausea
  • Abdominal Pain
• Hypersensitivity:
  • Fixed Drug Eruption
  • Anaphylaxis
• Neuromuscular & Skeletal:
  • Back Pain

Contraindication to Glucocerebrosidase (Taliglucerase alfa Injection):

• In the US labeling for taliglucerase alfa, there are no listed contraindications, meaning there are no specific situations where the medication should not be used.
• In the Canadian labeling, it's noted that severe hypersensitivity to taliglucerase alfa or any component of the formulation is a contraindication. This means that if someone has a severe allergic reaction to taliglucerase alfa or any ingredient in the medication, they should not use it.

Warnings and precautions

Formation of antibodies:

• Some patients treated with taliglucerase alfa have developed IgG anti-drug antibodies (ADA), although the impact of this on health isn't clear.
• It's uncertain whether having these antibodies increases the risk of infusion reactions.
• However, patients who have immune or infusion reactions to taliglucerase alfa, or who have experienced immune responses to other enzyme replacement therapies and are switching to taliglucerase alfa, should be watched for ADA development.
• Monitoring for these antibodies helps doctors assess potential risks and tailor treatment accordingly.

CNS effects

• Patients taking taliglucerase alfa may experience dizziness and fatigue as side effects.
• It's important for consultant to advise patients about these potential effects and to caution them against engaging in activities that require focus and coordination, such as driving or operating machinery, until they know how the medication affects them individually.
• This helps ensure the safety of patients and those around them.

Hypersensitivity/anaphylactoid reactions

• Hypersensitivity and anaphylactoid reactions, including severe allergic responses like anaphylaxis, can happen with taliglucerase alfa, even up to three hours after starting the infusion.
• It's crucial to have medical support ready in case of such reactions.
• The management of these reactions depends on their severity; for mild reactions, slowing or temporarily stopping the infusion and providing premedication like antihistamines or corticosteroids may help.
• Preemptive treatment before subsequent infusions may also prevent reactions.
• Patients should be closely monitored during and after infusion.
• If severe reactions occur, the infusion should be stopped immediately, and appropriate treatment initiated.
• Reintroducing the medication should be done cautiously after such reactions.

Monitoring parameters:

Blood Tests:

• Keep an eye on hemoglobin levels and platelet counts.

Enzyme Levels:

• Check angiotensin converting enzyme, tartrate-resistant acid phosphatase, and chitotriosidase levels.

Anti-Drug Antibodies:

• Monitor for IgG anti-drug antibody formation, especially in patients who had immune or infusion reactions to enzyme replacement therapy before.

Organ Sizes:

• Measure liver and spleen volumes to track any changes.

Bone Health:

• Assess bone density to watch for any changes.

Heart Health:

• Conduct ECG and echocardiogram tests, along with chest x-rays, to ensure heart health.

How to administer Glucocerebrosidase (Taliglucerase alfa Injection)?

Administering Taliglucerase Alfa via IV

• Infusion Time: Infuse over at least 60 minutes, typically lasting between 60 to 120 minutes.
• Infusion Set: Use a low protein-binding infusion set with a 0.2 micron in-line filter.
• Initiation Rate: Begin infusion at a rate of 1.2 mL per minute.
• Adjustment: You can increase the rate, but do not exceed 2.2 mL per minute based on how well the patient tolerates it.

Mechanism of action of Glucocerebrosidase (Taliglucerase alfa Injection):

• Taliglucerase alfa is a type of enzyme replacement therapy used to treat Gaucher's disease.
• It's made through recombinant DNA technology using plant (carrot) cell culture.
• This medication acts like glucocerebrosidase, an enzyme that's lacking in Gaucher's disease.
• Glucocerebrosidase normally helps break down a fatty substance called glucocerebroside into glucose and ceramide.
• When this enzyme is missing, glucocerebroside builds up, causing problems like enlarged liver and spleen, anemia, and low platelet counts.
• Taliglucerase alfa steps in to help reduce these symptoms by replacing the missing enzyme, improving liver and spleen size, and addressing anemia and thrombocytopenia.

Distribution Volume (V):

• Pediatric Patients: 8.8 to 14.9 liters
• Adults: 10.7 to 11.7 liters

Half-life of Elimination:

• Pediatric Patients: 33 to 37 minutes
• Adults: 19 to 29 minutes
  • Note: Half-life can increase with higher doses.

International Brand Names of Taliglucerase alfa:

• Eleliso
• Elelyso
• Eleyso
• Uplyso

Taliglucerase alfa Brand Names in Pakistan:

No Brands Available in Pakistan.