Patient demographics and baseline characteristics
| Parameter | Pegunigalsidase alfa | Agalsidase beta | Overall (n=77) | P value* | ||||
| Males (n=29) | Females (n=23) | Overall (n=52) | Males (n=18) | Females (n=7) | Overall (n=25) | |||
| Age, years | 0.60 | |||||||
| Mean±SD | 42.6±11.5 | 45.6±8.3 | 43.9±10.2 | 46.5±6.9 | 41.7±14.5 | 45.2±9.6 | 44.3±10.0 | |
| Sex, n (%)† | 29 (56) | 23 (44) | – | 18 (72) | 7 (28) | – | M 47 (61) F 30 (39) | 0.19 |
| eGFR, mL/min/1.73 m2‡ | ||||||||
| Mean (SE) | 71.6 (4.4) | 75.8 (3.0) | 73.5 (2.8) | 69.2 (5.0) | 86.9 (5.3) | 74.2 (4.2) | 73.7 (2.3) | 0.82 |
| Median | 70.2 | 75.5 | 73.5 | 71.8 | 88.2 | 74.9 | 74.5 | |
| Min, max | 30.2, 125.9 | 47.2, 107.1 | 30.2, 125.9 | 34.1, 106.3 | 65.8, 107.6 | 34.1, 107.6 | 30.2, 125.9 | |
| eGFR slope, mL/min/1.73 m2/year§ | 0.37 | |||||||
| Mean (SE) | −8.7 (1.5) | −7.2 (1.0) | −8.0 (0.9) | −7.8 (1.1) | −9.4 (1.0) | −8.3 (0.9) | −8.1 (0.7) | |
| Median | −7.3 | −6.5 | −6.7 | −7.3 | −8.3 | −7.8 | −7.3 | |
| Min, max | −30.5, 6.3 | −19.2,–1.6 | −30.5, 6.3 | −20.3,–2.8 | −13.9,–6.9 | −20.3,–2.8 | −30.5, 6.3 | |
| UPCR, n (%) | 0.52 | |||||||
| UPCR≤0.5 g/g | 15 (52) | 21 (91) | 36 (69) | 13 (72) | 7 (100) | 20 (80) | 56 (73) | |
| 0.5<UPCR <1 g/g | 8 (28) | 1 (4) | 9 (17) | 2 (11) | 0 | 2 (8) | 11 (14) | |
| UPCR≥1 g/g | 6 (21) | 1 (4) | 7 (14) | 3 (17) | 0 | 3 (12) | 10 (13) | |
| Treatment with ACEi or ARBs, n (%) | 17 (59) | 9 (39) | 26 (50) | 15 (83) | 1 (14) | 16 (64) | 42 (55) | 0.22 |
| Positive ADA status¶, n (%) | 18 (62) | 0 | 18 (35) | 8 (44) | 0 | 8 (32) | 26 (34) | 0.82 |
| Positive for neutralising antibodies, n (%)** | 17 (59) | 0 | 17 (33) | 7 (39) | 0 | 7 (28) | 24 (31) | 0.8 |
| Length of previous agalsidase beta treatment, years†† | 0.25 | |||||||
| Mean±SD | 6.4±4.9 | 4.2±2.1 | 5.4±4.0 | 6.6±3.4 | 6.1±3.7 | 6.4±3.4 | 5.8±3.8 | |
*P values were calculated between treatment arms for age and length of previous agalsidase beta treatment with t-test; for sex, UPCR category, ACEi/ARB treatment, and ADA status by Pearson χ2 test; for eGFR and eGFR slope by Wilcoxon; for neutralising antibodies by Fisher’s exact test.
†Percentage calculated out of total number of patients per treatment arm.
‡Inclusion criteria specified patients have eGFR of 40–120 mL/min/1.73 m2 at screening visit; eGFR at baseline visit (presented here) was outside of this range for some patients. Normal range 90–120 mL/min/1.73 m2.46
§eGFR slope at baseline was based on historical, screening, and baseline serum creatinine measurements and was more positive than −2 mL/min/1.73 m2/year at baseline for some patients. eGFR slope as negative as −1 mL/min/1.73 m2/year is considered normal for patients age ≥40 years.46
¶All patients were evaluated for the presence of antidrug IgG antibodies to their assigned drug at baseline.
**Percentage calculated out of total number of patients of the respective sex per treatment arm.
††Last continuous agalsidase beta treatment.
ACEi, angiotensin-converting enzyme inhibitors; ADAs, antidrug antibodies; ARBs, angiotensin II receptor blockers; eGFR, estimated glomerular filtration rate; F, female; M, male; UPCR, urine protein creatinine ratio.