Table 1.
Efficacy of oral migalastat in ERT-naive patients with Fabry disease at the end of the initial 6-month double-blind period of a randomized phase 3 trial (FACETS)
| Treatment group | ≥ 50% ↓ in no. of GL-3 inclusions/KICa [% of pts] (no. of pts) | Mean change from BL in no. of GL-3 inclusions/KICb,c (no. of pts) | Mean change from BL in plasma lyso-Gb3 levelsb [ng/mL] (no. of pts) |
|---|---|---|---|
| ITT pts | mITT ptsd | mITT ptsd | |
| Migalastat | 41 (32) | − 0.25* (25) | − 11.20** (18) |
| Placebo | 28 (32) | 0.07 (20) | 0.60 (13) |
Pts (aged 16–74 years) received migalastat 123 mg once every other day or placebo [29]
BL baseline, GL-3 globotriaosylceramide, ITT intent-to-treat, KIC kidney interstitial capillary, lyso-Gb3 globotriaosylsphingosine, mITT modified ITT, pts patients, ↓ indicates decrease
*p = 0.008, **p = 0.003 vs. placebo
aPrimary endpoint
bBL values normalized to zero
cPost-hoc analysis
dmITT population: 45 pts had BL and post-BL renal biopsy data available and 31 pts consented to plasma lyso-Gb3 analyses