Table 3.
Number of events attributable to Fabry disease up to month 18 in the Japanese patient population and the overall patient population (mITT population) of the ATTRACT study
| Event, n (%) | Japanese | Overall | ||
|---|---|---|---|---|
| Migalastat (n = 5) | ERT (n = 1) | Migalastat (n = 34) | ERT (n = 18) | |
| Renal | 1 (20.0)a | 0 | 8 (23.5) | 6 (33.3) |
| Cardiac | 0 | 1 (100.0)b | 2 (5.9) | 3 (16.7) |
| Cerebrovascular | 0 | 0 | 0 | 1 (5.6) |
| Death | 0 | 0 | 0 | 0 |
| Total | 1 (20.0) | 1 (100.0) | 10 (29.4) | 8 (44.4) |
ERT enzyme replacement therapy, mITT modified intention-to-treat population
Patients in the mITT population are those with amenable GLA mutations who were assessed for baseline and postbaseline efficacy measures
a> 33% increase of 24-h urinary protein excretion compared with baseline and 24-h urinary protein excretion ≥ 300 mg
bNew York Heart Association class III/IV congestive heart failure