Clinical study results

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Clinical study results

There are over 129 patient-years* of data in combined clinical trials of ELELYSO® (taliglucerase alfa) for injection.

*Patient-years=number of patients multiplied by the average amount of time on ELELYSO.

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In adults: 9-month study

Based on a study of previously untreated adults with Type 1 Gaucher disease, adults taking ELELYSO showed improvement after 9 months of treatment across a range of symptoms. This included a significant improvement in the main goal of the study, reduction in spleen size. It also included a significant reduction in liver size, and increased hemoglobin level and platelet count.

Data are from a 9-month pivotal trial of 31 patients with Type 1 Gaucher disease. The trial compared the effectiveness and safety of 2 different doses of ELELYSO. Patients were previously untreated and randomly chosen to receive either 30 or 60 Units of ELELYSO per kilogram of body weight every other week. The recommended dosage in treatment-naïve adult patients is 60 Units per kilogram of body weight every other week. ELELYSO 30 Units per kilogram of body weight is not a recommended dosage.

Adults taking 60 Units of ELELYSO per kilogram of body weight after 9 months experienced, on average:

  • Mean Spleen Size (main study goal) Decreased by 35%
  • Mean Hemoglobin LevelIncreased by 19%
  • Mean Liver SizeDecreased by 12%
  • Mean Platelet CountIncreased by 64%

In adults: extension study

In an extension of the initial 9-month study of ELELYSO, 26 previously untreated adult patients were followed for an additional 15 months in double-blind fashion. They showed continued improvement on treatment with ELELYSO after the initial study for a total treatment duration of 24 months.

Adults taking 60 Units of ELELYSO per kilogram of body weight after a total of 24 months experienced, on average:

  • Mean Spleen Size (main study goal)Decreased by 55%
  • Mean Hemoglobin LevelIncreased by 21%
  • Mean Liver SizeDecreased by 19%
  • Mean Platelet CountIncreased by 104%

In adults: 12-month continuation of the extension study

Following the 15 months of blinded treatment in the extension study, 23 of the 26 patients who continued treatment in an open-label fashion demonstrated continued improvements in the above clinical parameters.

Double-blind means that neither the researchers nor the patients knew which dose (30 or 60 Units of ELELYSO per kilogram of body weight) the patients were receiving.

Open label means that both the researchers and the patients knew which dose (30 or 60 Units of ELELYSO per kilogram of body weight) the patients were receiving.

In children: 12-month study

In a 12-month study in children with Type 1 Gaucher disease who were previously untreated, children taking ELELYSO showed improvements in the clinical endpoints of the study, which included a reduction of spleen and liver size and an increase in platelet count.§

Children taking 60 Units of ELELYSO per kilogram of body weight after 12 months experienced, on average:

  • Mean Spleen SizeDecreased by 41%
  • Mean Platelet CountIncreased by 74%
  • Mean Liver SizeDecreased by 14%

The pediatric labeling of ELELYSO does not include change in hemoglobin level, which was the main goal of the 12-month pediatric study. Approval for the pediatric indication was supported by clinical data in adults and children and by data showing how ELELYSO is absorbed and distributed in children.

§Data are from a 12-month clinical trial of 9 children ages 2-13 with Type 1 Gaucher disease. The trial compared the effectiveness and safety of 2 different doses of ELELYSO. Patients were previously untreated and randomly chosen to receive either 30 or 60 Units of ELELYSO per kilogram of body weight every other week. The recommended dosage in treatment-naïve pediatric patients 4 years of age and older is 60 Units per kilogram of body weight every other week. ELELYSO 30 Units per kilogram of body weight is not a recommended dosage. There are insufficient data to inform dosing in patients less than 4 years of age.

In adults and children: 9-month switch study

A 9-month study was done to assess the safety and effectiveness of ELELYSO in 26 adults and 5 children switching from Cerezyme®|| (imiglucerase for injection) to ELELYSO. People in the study were switched from Cerezyme to ELELYSO at the same dosage.

Mean spleen size, liver size, hemoglobin level, and platelet count remained stable in both adults and children switched from imiglucerase to ELELYSO over the 9-month period.

||Cerezyme is a registered trademark of Genzyme Corporation.

Based on a 9-month, multicenter, open-label clinical trial of 26 adults and 5 children switching from Cerezyme to ELELYSO. All patients were clinically stable at the time of the clinical trial and had a stable biweekly dosage of imiglucerase for at least 6 months prior to enrollment.

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