Open-Label Phase 3 Long-Term Safety Study of Migalastat

Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)

Time frame · Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months.

An adverse event (AE) was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at the time of reporting; visits typically occurred every 6 months. A TEAE was defined as an AE starting on or after the first study drug administration date. Serious AEs were life-threatening or resulted in death, persistent or significant incapacitation, inpatient or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: minimal discomfort, does not interfere with normal everyday activities; Moderate: sufficiently discomforting, interferes with normal everyday activities; Severe: prevents normal everyday activities. The number of participants experiencing TEAEs is presented for those who received migalastat treatment. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.

Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)

Time frame · Baseline, Every 6 m until the End of Study (42 m)

The annualized rate of change of the eGFR was assessed per participant by the slope of the simple linear regression between the observed values and the assessment times. It was calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (eGFR \[CKD-EPI\]) and the Modification of Diet in Renal Disease (MDRD) equation (eGFR \[MDRD\]). The equations are as follows: eGFR \[MDRD\] = 175 \* (Serum Creatinine)\^-1.154 \* (Age)\^-0.203 \* 1.212 (if black or African American) \* 0.742 (if female); eGFR \[CKD-EPI\] = 141 \* min(serum creatinine/kappa,1)\^alpha \* max(serum creatinine/kappa, 1)\^-1.209 \* 0.993\^age \* 1.1018(if female) \* 1.159(if black or African American), where kappa is 0.7 for females and 0.9 for males, alpha is -0.329 for females and -0.411 for males, min is minimum of serum creatinine/kappa or 1, and max is the maximum of serum creatinine/kappa or 1. The number of participants with at least a Baseline and a post-Baseline value are presented.