TOTAL | Phase 2 Untreated | ENGAGE Untreated | ENCORE ERT Switch | EDGE Mostly ERT Switch | |
---|---|---|---|---|---|
Eliglustat-treated patients | 393 | 26 | 40 | 157 | 170 |
Patient-years of treatment exposure | 1400 | 169 | 154 | 511 | 566 |
Mean duration of treatment (years) | 3.6 | 6.5 | 3.9 | 3.3 | 3.3 |
Remained in trial until completion or availability of commercial drug, n (%) | 319 (81.2) | 19 (73.1) | 34 (85.0) | 129 (82.2) | 137 (80.6) |
Switched to commercial eliglustat, n | 80 | 0 | 7 | 52 | 21 |
Previously treated with enzyme replacement therapy, n (%) | 305 (77.6) | 0 (0) | 0 (0) | 157 (100.0) | 148 (87.1) |
Active withdrawals, n (%) | 74 (18.8) | 7 (26.9) | 6 (15.0) | 28 (17.8) | 33 (19.4) |
Due to any AEa | 25 (6.4) | 3 (11.5) | 0 (0) | 12 (7.6) | 10 (5.9) |
Wished to withdraw | 25 (6.4) | 1 (3.8) | 5 (12.5) | 8 (5.1) | 11 (6.5) |
Due to pregnancy | 15 (3.8) | 3 (11.5) | 1 (2.5) | 4 (2.5) | 7 (4.1) |
Due to noncompliance | 3 (0.8) | 0 (0) | 0 (0) | 0 (0) | 3 (1.8) |
Lost to follow-up | 3 (0.8) | 0 (0) | 0 (0) | 1 (0.6) | 2 (1.2) |
Other | 3 (0.8) | 0 (0) | 0 (0) | 3 (1.9) | 0 (0) |
Any patient with an AE, n (%) | 373 (94.9) | 26 (100.0) | 36 (90.0) | 147 (93.6) | 164 (96.5) |
Number of events, n | 4814 | 348 | 559 | 2153 | 1754 |
Mild, n (%) | 3551 (73.7) | 251 (72.1) | 448 (80.1) | 1590 (73.9) | 1262 (71.9) |
Moderate, n (%) | 1117 (23.2) | 91 (26.1) | 107 (19.1) | 501 (23.2) | 418 (23.8) |
Severe, n (%) | 146 (3.0) | 6 (1.7) | 4 (0.7) | 62 (2.9) | 74 (4.2) |
Any patient with treatment-relatedb AE, n (%) | 196 (49.9) | 10 (38.5) | 22 (55.0) | 83 (52.9) | 81 (47.6) |
Number of treatment-related AEs, n (% of AEs) | 682 (14.2) | 20 (5.7) | 94 (16.8) | 314 (14.6) | 254 (14.5) |
Any patient with serious AE, n (%) | 77 (19.6) | 5 (19.2) | 5 (12.5) | 27 (17.2) | 40 (23.5) |
Number of serious AEs, n | 103 | 8 | 7 | 28 | 60 |
Mild, n (%) | 17 (17) | 3 (38) | 4 (57) | 3 (11) | 7 (12) |
Moderate, n (%) | 37 (36) | 1 (13) | 3 (43) | 9 (32) | 24 (40) |
Severe, n (%) | 49 (48) | 4 (50) | 0 (0) | 16 (57) | 29 (48) |
Any patient with treatment-related serious AE, n (%) | 8 (2.0) | 1 (3.8) | 1 (2.5) | 2 (1.3) | 4 (2.4) |
Deaths, n (%) | 2 (0.5) | 0 (0) | 0 (0) | 0 (0) | 2 (1.2) |