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Switch Patient Study (ENCORE)
Cerdelga is the ONLY first-line oral therapy for most adults with Gaucher disease type 1, indicated for the long-term treatment of both naive and switch patients.1,2
The efficacy and safety of Cerdelga in patients previously treated with enzyme replacement therapy (ERT) was studied in a phase 3 ENCORE trial. ENCORE included a primary and open-label extension phase of Cerdelga in which patients were evaluated at 12 months and 2 years, and were observed for up to 4 years. Explore the results below.1,5,8
Key study design parameters (primary and open-label extension phases)1,5,6,8
Switch with confidence. Switch to Cerdelga.
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.1,2
After 1 year of treatment, ENCORE study results showed noninferiority to imiglucerase; 85% of Cerdelga patients met the primary composite endpoint compared to 94% in the imiglucerase group. The lower bound of the 95% CI (-17.6%) was within the prespecified noninferiority margin of -25%.1,5
There were no clinically meaningful differences between patients receiving Cerdelga vs imiglucerase (ERT), for any of the 4 parameters of the composite endpoint.1,5
Disease stability maintained in patients switched from ERT to Cerdelga1,5
Of the patients who did not meet stability criteria for the individual
components, 12 of 15 Cerdelga patients and 3 of 3 imiglucerase patients remained
within therapeutic goals for Gaucher disease type 1.1
Per-protocol population. Note: Error bars represent exact 95% CIs around the proportion.
*Spleen percentages are based on the total number of nonsplenectomized patients in each treatment group.
Adapted from Cox et al. Lancet. 2015.
Maintained stability in spleen and liver volume for up to 12 months1,5,6
Maintained stability in hemoglobin level and platelet count for up to 12 months1,5,6
Baseline is defined as before the first dose of study medication in the primary analysis phase. MN=multiples of normal.
All patients who completed the 12-month primary analysis period were allowed to participate in the open-label extension (OLE) phase and receive Cerdelga. Of the 159 patients in the primary analysis phase, 152 entered the extension phase (101/102 patients in the Cerdelga arm and 51/52 patients in the imiglucerase arm continued in the OLE phase and switched to Cerdelga).6,8 See the results from the OLE phase below.
Maintained stability in visceral and hematologic disease parameters for up to 2 years in the ENCORE study.1
Mean changes from baseline in individual components of the primary endpoint composite score8
Patients were followed for up to 4 years of Cerdelga treatment. Due to decreasing sample size, results after the 2-year analysis require cautious interpretation.
Percentage changes from baseline were calculated by dividing the LS mean change from baseline for each year (as reported in Cox et al, Blood, 2017) by the baseline value and multiplying by 100.
Baseline was defined as the last available assessment before Cerdelga treatment initiation (day 1 for patients originally randomized to Cerdelga and week 52 + 1 day for patients originally randomized to imiglucerase).
LS=least-square; CI=confidence interval; MN=multiples of normal.
Patients can be switched to Cerdelga in as little as 24 hours after their last ERT infusion.1
Primary Analysis Period
|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)|
|12 (11)||1 (2)|
|11 (10)||0 (0)|
|Dizziness||8 (9)||0 (0)|
|Asthenia||8 (9)||0 (0)|
|Cough||7 (7)||0 (0)|
|Dyspepsia||7 (7)||2 (1)|
|7 (7)||0 (0)|
|Constipation||5 (5)||0 (0)|
|Palpitations||5 (5)||0 (0)|
|Rash||5 (5)||0 (0)|
*ENCORE was not designed to support comparative claims for Cerdelga for the adverse reactions reported in this table.