The Medical Letter on Drugs and Therapeutics
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In Brief: Taliglucerase (Elelyso) for Gaucher Disease
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 Select a term to see related articles  Cerezyme   Elelyso   Gaucher disease   Imiglucerase   taliglucerase   Velaglucerase   Vpriv 

The FDA has approved taliglucerase alfa (ta lee gloo´ se rays; Elelyso – Pfizer/Protalix), a recombinant form of glucocerebrosidase, for treatment of adults with Type 1 Gaucher disease. These patients have a genetic deficiency of the lysosomal enzyme glucocerebrosidase that leads to accumulation of glucosylceramide in the lysosomes of reticuloendothelial cells, primarily in the liver, spleen and bone marrow.1

Taliglucerase is the third form of the enzyme to become available in the US. Imiglucerase (Cerezyme) and velaglucerase (Vpriv) are produced in mammalian cell lines. Taliglucerase is produced using carrot plant root cells, which is less costly, according to the manufacturer.

In one trial, taliglucerase significantly reduced spleen volume and increased serum hemoglobin.2 In a trial in patients receiving chronic imiglucerase therapy, summarized in the package insert, clinical parameters remained stable following a switch to an equal dose of taliglucerase.

All 3 of these enzymes are usually given as an IV infusion over 1-2 hours every 2 weeks. Infusion reactions are common. Anaphylaxis and development of IgG anti-drug antibodies have been reported. For 1 year's treatment of a 70-kg patient at 60 units/kg every 2 weeks, taliglucerase will cost $324,870, imiglucerase $432,978 and velaglucerase $368,550.3

1. TM Cox. Gaucher disease: clinical profile and therapeutic developments. Biologics 2010; 4:299.

2. A Zimran et al. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood 2011; 118:5767.

3. Wholesale acquisition cost (WAC). Source: PricePointRx™. Reprinted with permission by FDB. All rights reserved. ©2012. http://www.firstdatabank.com/support/drug-pricing-policy.aspx. Accessed June 20, 2012. Actual retail prices may be higher.

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