Drug information of taliglucerase alfa

Mechanism of effect

Taliglucerasealfa is different from human glucocerebrosidase by two amino acids at the N terminal and up to 7 amino acids at the C terminal. This recombinant enzyme allows the hydrolysis reaction of glucocerebroside to glucose and ceramide that naturally occurs in healthy individuals.

Pharmacodynamic

Patient's with Type 1 Gaucher disease have a long-term deficiency in the enzyme, glucocerebrosidase. Taliglucerasealfa is a modified form of glucocerebrosidase and is provided to counter this enzyme deficiency, resulting in smaller liver and spleen size, and improved thrombocytopenia and anemia.

Pharmacokinetics

Vd: 7.3-11.7 L

Half-life: 18.9-28.7 minutes

Systemic clearance: 20 L/hr (for 60 units/kg dose)

Drug indications

Gaucher's Disease Type 1 , Gaucher's Disease Type 1 and 3

Dosage

Adult

Gaucher Disease

60 units/kg IV q2weeks initially

Pediatric

Gaucher Disease

<4 years: Safety and efficacy not established

≥4 years: 60 units/kg IV q2weeks initially

Side effects

Headache , abdominal pain , vomiting , allergic reaction , flushing , vertigo , urticaria , itching , tiredness

Alerts

• Anaphylaxis reported; if anaphylaxis occurs, discontinue infusion immediately and initiate appropriate treatment
• Allergic and infusion reactions are common and include headache, chest pain or discomfort, asthenia, fatigue, urticaria, erythema, increased blood pressure, back pain, arthralgia, and flushing; if allergic or infusion reactions occur, decrease the infusion rate or temporarily stop the infusion; antihistamines and/or antipyretics may be administered; medical support should be readily available when taliglucerasealfa administered; observe patients closely for an appropriate period of time after administration; reactions have occurred up to 3 hr after the start of infusion
• Consider risks and benefits of re-administering taliglucerasealfa in patients who have experienced severe reaction associated with taliglucerasealfa; use caution upon rechallenge; appropriate medical support should be readily available
• As with all therapeutic proteins, development of IgG anti-drug antibodies (ADA) has been reported (53%); unknown if presence of ADA is associated with higher risk of infusion reactions or immunogenicity
• Monitor patients that have had an immune response to other enzyme replacement therapies or who are switching to taliglucerasealfa; unknown if these patients are at higher risk of infusion reactions
• Dizziness and fatigue reported; caution patients against performing dangerous tasks that require mental alertness, including driving heavy machinery

Pregnancy level

B

Related drugs

Imiglucerase , Velaglucerase alfa