General Information

Elelyso (taliglucerase alfa) is a hydrolytic lysosomal glucocerebroside-specific enzyme that functions as a long-term enzyme replacement therapy (ERT).

Elelyso is specifically indicated for the treatment of Type 1 Gaucher disease in adults.

Elelyso is supplied as an infusion designed for intravenous administration. The recommended initial dose of the drug is 60 units/kg administered every other week for 60-120 minutes. Dosage adjustments may be made based on achievement and maintenance of particular therapeutic goals.

Clinical Results

FDA Approval
FDA Approval of Elelyso was based on the results of two 9 month multi-center trials.
Trial One
This double-blind, randomized study enrolled 31 adults with Gaucher disease-related enlarged spleens and thrombocytopenia who were ERT-naïve. Sixteen subjects had enlarged livers and 10 had anemia at baseline. Subjects were randomized to be treated with Elelyso doses of either 30 units/kg (n=15) or 60 units/kg (n=16). The primary endpoint of change in spleen volume (SD) was observed in MRI at nine months measured by mean % body weight (%BW) and multiples of normal (MN) spleen volumes. Liver volumes and hemoglobin concentrations were also monitored. Subjects in the 30 units/kg and treatment arm demonstrated changes of -0.9 (0.4) %BW mean (SD) and -4.5 (2.1) MN mean (SD). Subjects treated with 60 units/kg demonstrated changes of -1.3 (1.1) %BW mean (SD) and -6.6 (5.4) MN mean (SD).
Trial Two
This open-label single arm study enrolled 25 subjects with Type 1 Gaucher disease who were switched to Elelyso from a 2-year minimum treatment duration of imiglucerase. Subjects were clinically stable and were administrered a stable biweekly dose of imiglucerase for at least 6 months prior to enrollment. Elelyso was administered at the same dosage level as the previous imiglucerase treatment, 11 units/kg to 60 units/kg biweekly. Organ volumes and hemotologic values remained stable on average during the 9 month treatment period.

Mechanism of Action

Elelyso (taliglucerase alfa) is a recombinant form of human Glucocerebrosidase (GCD). It is a lysosomal glucocerebroside-specific enzyme that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. Subjects with Gaucher disease do not produce enough GCD, which leads to the accumulation of glucocerebroside in the spleen, liver, and bone marrow.