Plasma lyso-Gb3 from baseline to 24 months by treatment arm and sex
| Plasma lyso-Gb3 (nM)* | Pegunigalsidase alfa | Agalsidase beta | ||||
| Male (n=29) | Female (n=23) | Overall (n=52) | Male (n=18) | Female (n=7) | Overall (n=25) | |
| Baseline, n | 29 | 23 | 52 | 18 | 7 | 25 |
| Mean (SE) | 40.40 (5.50) | 8.35 (0.68) | 26.22 (3.78) | 42.43 (8.71) | 5.69 (1.10) | 32.14 (7.08) |
| Median | 30.7 | 8.4 | 15.2 | 23.7 | 4.4 | 17.6 |
| Min, max | 0.8, 143.9 | 2.8, 16.2 | 0.8, 143.9 | 8.9, 142.0 | 2.1, 10.4 | 2.1, 142.0 |
| 24 months, n | 25 | 21 | 46 | 15 | 7 | 22 |
| Mean (SE) | 46.88 (6.34) | 8.19 (0.95) | 29.22 (4.48) | 26.17 (4.33) | 5.66 (1.06) | 19.65 (3.60) |
| Median | 34.4 | 8.9 | 18.8 | 20.5 | 4.9 | 15.3 |
| Min, max | 3.2, 139.4 | 2.4, 22.0 | 2.4, 139.4 | 6.2, 71.2 | 1.5, 9.7 | 1.5, 71.2 |
| Change from baseline to 24 months, n† | 25 | 21 | 46 | 15 | 7 | 22 |
| Mean (SE) | 5.90 (2.41) | 0.19 (0.46) | 3.30 (1.38) | −12.80 (6.93) | −0.03 (0.27) | −8.74 (4.85) |
| Median | 5.3 | 0.1 | 1.15 | −2.40 | −0.30 | −1.50 |
| Min, max | −32.2, 32.7 | −4.0, 5.8 | −32.2, 32.7 | −102.3, 2.4 | −0.7, 0.9 | −102.3, 2.4 |
*Normal range ≤2.4 nM.
†Data from five patients in the pegunigalsidase alfa arm and one in the agalsidase beta arm are missing due to early termination; one and two patients, respectively, are missing data due to missed visits.
Lyso-Gb3, globotriaosylsphingosine.