Important Safety Information: Certain patients should not use CERDELGA based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Do not use CERDELGA if you are an Extensive Metabolizer (EM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or... View more

Study 1: Untreated Patient Study

Cerdelga is the ONLY first-line oral therapy indicated for the long-term
treatment of most adults with Gaucher disease type 1.

Tamara and Nate, USA,
patients with Gaucher disease type 1 treated with Cerdelga

View Indications and Usage

Study 1 Overview

Study 1 involved 40 adult patients with Gaucher disease type 1 who had not been on treatment or who were no longer receiving treatment. Spleen size, liver size, platelet levels, and hemoglobin levels were evaluated.

About study 1

Patients with Gaucher disease type 1

40 patients with Gaucher disease type 1 who were at least 16 years of age.

Gaucher disease type 1 doctor 

No study personnel, including the doctors, knew which patients were taking Cerdelga and which were taking placebo.

Duration of the primary analysis  

The duration of the primary analysis phase was 9 months. 20 patients were taking Cerdelga, and 20 taking placebo.

Some patients continued in the study for up to 2 years. After the 9-month primary analysis phase, patients in the placebo group had the opportunity to switch to Cerdelga. 39 of 40 patients continued in the study.

Cerdelga is the ONLY first-line oral treatment for most adult patients with Gaucher disease type 1 that significantly improved organ and blood parameters in previously untreated patients.

Talk to Your Doctor about Starting Cerdelga

Explore the study results below

At 9 months, compared with the placebo, Cerdelga showed:

Cerdelga short-term test results in the spleen and liverCerdelga short-term test results in hemoglobin and platelet levels

When evaluated at 2 years:

In the long-term, open-label extension phase, patients taking Cerdelga showed the following changes in spleen and liver size and in hemoglobin and platelet levels

Cerdelga long-term test results in the spleen and liverCerdelga long-term test results in hemoglobin and platelet levels

Patients continued to be observed for up to 4.5 years.

Study 1 Safety

The most common side effects
(occurring in ≥10% patients)
with Cerdelga (eliglustat)

Side effect Cerdelga
(n=20)		 Placebo
(n=20)
Number of patients (%)
Joint Pain 9 (45%) 2 (10%)
Headache 8 (40%) 6 (30%)
Migraine 2 (10%) 0 (0%)
Flatulence 2 (10%) 1 (5%)
Nausea 2 (10%) 1 (5%)
Mouth Pain 2 (10%) 1 (5%)
  • • 0 patients discontinued the study due to side effects in the primary analysis period
  • • No new safety signals were observed in the long-term extension period

Talk to your doctor about starting treatment with Cerdelga.