Median eGFR slope and 95% CI model* (ITT population)—by treatment arm, sex and ADA status
| ITT population median eGFR slope | Pegunigalsidase alfa (n=52)† | Agalsidase beta (n=25)‡ | Difference between arms |
| Baseline, mL/min/1.73 m2/year | |||
| Overall | −6.70 | −7.84 | – |
| Male | −7.25 | −7.25 | – |
| Female | −6.45 | −8.31 | – |
| ADA-positive | −5.75 | −6.08 | – |
| ADA-negative | −7.10 | −7.84 | – |
| 24 months, mL/min/1.73 m2/year (95% CI) | |||
| Overall | −2.51 (−3.79, –1.24) | −2.16 (−3.81, –0.51) | −0.36 (−2.44§, 1.73) |
| Male | −3.44 (−5.38, –1.50) | −2.01 (−3.98, –0.04) | −1.43 (−3.96, 1.10) |
| Female | −1.15 (−3.11, 0.81) | −2.79 (−6.28, 0.70) | 1.64 (−2.56, 5.84) |
| ADA-positive | −2.51 (−5.28, 0.25) | −2.16 (−6.25, 1.93) | −0.36 (−5.16, 4.45) |
| ADA-negative | −2.22 (−4.02, –0.43) | −2.16 (−4.06, –0.26) | −0.07 (−2.41, 2.27) |
*To determine non-inferiority, the annualised median eGFR slopes were analysed by quantile regression using SAS PROC QUANTREG to obtain the corresponding 95% CI; non-inferiority was declared if the lower limit of the CI for the treatment difference (pegunigalsidase alfa – agalsidase beta) was ≥ −3.0 mL/min/1.73 m2/year.
†Baseline: males (n=29), females (n=23), ADA-positive (n=18), ADA-negative (n=34); number of subjects considered in the model at 24 months: overall (n=51), males (n=28), females (n=23), ADA-positive (n=17), ADA-negative (n=34).
‡Baseline: males (n=18), females (n=7), ADA-positive (n=8), ADA-negative (n=17); number of subjects considered in the model at 24 months: overall (n=25), males (n=18), females (n=7), ADA-positive (n=8), ADA-negative (n=17).
§Value above the predefined non-inferiority margin.
ADA, antidrug antibody; eGFR, estimated glomerular filtration rate; ITT, intent-to-treat.