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 2: ERT Switch 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 2 Overview

Study 2 involved 159 adult patients with Gaucher disease type 1 who were previously treated with ERT. The main goal of Study 2 was to see if patients switching to Cerdelga did as well as patients taking an ERT. Patients needed to remain stable on all of the following measures: spleen size, liver size, platelet levels, and hemoglobin levels.

About study 2

Patients with Gaucher disease type 1

159 patients with Gaucher disease type 1 who were all 18 years of age or older

IV solution bag

Before the study, people had been stable on ERT

Cerdelga capsule

Randomized 2:1 Cerdelga to imiglucerase

Duration of the primary analysis

The duration of the primary analysis phase was 12 months. 106 patients were taking Cerdelga, and 53 were taking imiglucerase.
Some patients continued in the study for up to 2 years. After the 12-month primary analysis phase, patients in the ERT group had the opportunity to switch to Cerdelga. 152 patients continued in the study and all took Cerdelga.

  • Most patients taking Cerdelga remained stable
  • Patients continued to be observed for up to 4 years

Explore the study results below

Cerdelga is the ONLY first-line oral treatment for most adults with Gaucher disease type 1 that maintained long-term disease stability in patients who were switched from ERT.

Study 2 successfully showed that Cerdelga is no less effective than ERT at 12 months

Study 2 successfully showed that Cerdelga is no less effective than ERT at 12 months in the primary analysis phase of the trial.

Patients on Cerdelga remained stable

After 12 months of treatment, patients on Cerdelga remained stable after switching to an ERT.

Liver, spleen, hemoglobin, platelets

There were no clinically meaningful differences between patients receiving Cerdelga versus ERT for any of the 4 measures.

2-year study

When evaluated at 2 years, in the long-term, open-label extension phase of the study, most patients who continued taking Cerdelga remained stable.

Study 2 Safety

The most common side effects
(occurring in >5% patients)
with Cerdelga (eliglustat)

Adverse Reaction, n (%) Cerdelga® (n=106) Imiglucerase (n=53)
Fatigue 15 (14%) 1 (2%)
Headache 14 (13%) 1 (2%)
Nausea 13 (12%) 0 (0%)
Diarrhea 13 (12%) 2 (4%)
Back pain 13 (12%) 3 (6%)
Pain in extremity 12 (11%) 1 (2%)
Upper abdominal pain 11 (10%) 0 (0%)
Dizziness 9 (8%) 0 (0%)
Asthenia 9 (8%) 0 (0%)
Cough 7 (7%) 2 (4%)
Dyspepsia 7 (7%) 1 (2%)
Gastroesophageal reflux disease 7 (7%) 0 (0%)
Constipation 5 (5%) 0 (0%)
Palpitations 5 (5%) 0 (0%)
Rash 5 (5%) 0 (0%)

*Trial 2 was not designed to support comparative claims for CERDELGA for the adverse reactions reported in this table.

  • • No new safety signals were observed in the long-term extension period

Interested in switching to Cerdelga? Talk to your Doctor.

You can switch in as little as 24 hours after your last ERT infusion.