Taliglucerase alfa (Elelyso) for the Treatment of Type 1 Gaucher disease- Pharmacology

ELELYSO (taliglucerase alfa) for injection, for intravenous use:

Taliglucerase alfa, is a hydrolytic lysosomal glucocerebroside-specific enzyme for intravenous infusion in  patients with 'Gaucher disease' (rare genetic disorder). The drug is a recombinant active form of the lysosomal enzyme, β-glucocerebrosidase, which is expressed in genetically modified carrot plant root cells cultured in a disposable bioreactor system (ProCellEx®). It is the first 'plant made' drug approved by the U.S Food and Drug administration (FDA).
β-Glucocerebrosidase (β-D-glucosyl-N-acylsphingosine glucohydrolase, E.C. is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide. 
ELELYSO is produced by recombinant DNA technology using plant cell culture (carrot). Purified taliglucerase alfa is a monomeric glycoprotein containing 4 Nlinked glycosylation sites (Mr = 60,800)

ELELYSO catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. In clinical trials, ELELYSO reduced spleen and liver size and improved anemia and thrombocytopenia.

First FDA US Approval: in 2012 for adults, in 2014 for pediatric use

ELELYSO™ (taliglucerase alfa) for injection is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease. 

60 Units/kg administered every other week as a 60-120 minute intravenous infusion.
Patients currently being treated with imiglucerase for 'Gaucher disease' can be switched to ELELYSO. Patients previously treated on a stable dose of imiglucerase are recommended to begin treatment with ELELYSO at that same dose when they switch from imiglucerase to ELELYSO.
Physicians can make dosage adjustments based on the achievement and maintenance of each patient’s therapeutic goals. Clinical trials have evaluated doses ranging from 11 Units/kg to 73 Units/kg every other week. 

For injection: lyophilized powder for reconstitution with diluent.
Available in 200 Unit single-use vials


Anaphylaxis has been observed in some patients treated with ELELYSO. If anaphylaxis occurs, immediately discontinue infusion and initiate appropriate treatment
Allergic and Infusion Reactions: 
The most commonly observed symptoms of infusion reactions (including allergic reactions) were headache, chest pain or discomfort, asthenia, fatigue, urticaria, erythema, increased blood pressure, back pain, arthralgia, and flushing. If allergic or infusion reactions occur, decreasing the infusion rate, temporarily stopping the infusion, or administering antihistamines and/or antipyretics is recommended. 

The most common adverse reactions during clinical studies were infusion reactions. Other commonly observed adverse reactions in >10% of patients were URTI/nasopharyngitis, pharyngitis/throat infection, headache, arthralgia, influenza/flu, UTI/pyelonephritis, back pain, extremity pain.