A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)

Time frame · Day 1 (after dosing) through Week 96

TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 96 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.

PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat

Time frame · 0 (predose), 0.5, 1, 2, 3, 4, 5, 6, 8, and 10 hr (postdose)

The AUC from time zero to 12 hours (hr) postdose (AUC0-12) was evaluated in plasma following a single dose of migalastat 25, 100, and 250 mg on Days 1, 15, and 29, respectively. In addition, AUC0-12 was assessed following multiple doses (14 days) of migalastat 25, 100, and 250 mg on Days 14, 28, and 42, respectively.

α-Galactosidase A (α-Gal A) Activity In Leukocytes At Baseline, Week 12, And Week 96

Time frame · Baseline, Week 12 (end of treatment period), Week 96 (end of extension period)

Leukocytes were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole \[nmol\] 4-MU/hr) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.