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
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.
159 patients with Gaucher disease type 1 who were all 18 years of age or older
Before the study, people had been stable on ERT
Randomized 2:1 Cerdelga to imiglucerase
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.
Study 2 successfully showed that Cerdelga is no less effective than ERT at 12 months in the primary analysis phase of the trial.
After 12 months of treatment, patients on Cerdelga remained stable after switching to an ERT.
There were no clinically meaningful differences between patients receiving Cerdelga versus ERT for any of the 4 measures.
When evaluated at 2 years, in the long-term, open-label extension phase of the study, most patients who continued taking Cerdelga remained stable.
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.