The following article features coverage from the 17th Annual WORLDSymposium meeting. Click here to read more of MPR’s conference coverage.

Long-term treatment with migalastat was associated with sustained reduction in left ventricular mass index (LVMi) in patients with Fabry disease, according to research presented the 17th Annual WORLDSymposium.

Fabry disease (FD) is a rare disorder caused by the accumulation of glycosphingolipid throughout the body, including the heart. Cardiac disease has been found to be a common cause of mortality in FD patients. Migalastat, an oral alpha-galactosidase A pharmacological chaperone, is approved to treat adult patients with FD as well as amenable variants.

Results from the phase 3 FACETS and ATTRACT trials evaluating migalastat demonstrated mean decreases in LVMi of -7.7g/m2 in treatment-naive FD patients and -3.8 g/m2 in enzyme replacement therapy-experienced FD patients after 24 and 30 months of therapy, respectively. “Here, we extended these findings and assess the long-term effects of migalastat on cardiac outcomes and safety in the international, phase 3 open-label extension study AT1001-042 (NCT02194985),” the study authors explained.

The extension study included a total of 84 adult FD patients who completed the FACETS, ATTRACT, or AT1001-041 study and continued to receive migalastat 150mg every other day. Echocardiography was utilized to assess LVMi, left ventricular (LV) ejection fraction, fractional shortening, as well as wall thickness. The average (standard deviation [SD]) age of the patients included in the study was reported to be 51.9 (12.3) years old and the mean (SD) duration of migalastat treatment was 2.7 (1.0) years. At baseline, the mean (SD) LVMi was reported to be 96.5 (36.6)g/m2, and 11.9% (10/84) of patients had a history of LV hypertrophy.

Findings revealed LVMi remained stable, with the mean (SD) change from baseline to the end of the study being -0.8 (11.8)g/m2. Male patients (n=8) experienced a -0.7 (11.5)g/m2 change, while female patients (n=16) experienced a -0.8 (12.4)g/m2 change. The authors also reported observing stable LV ejection fraction, fractional shortening, and wall thickness values during their assessment. Additionally, the majority of treatment-emergent adverse events were found to be mild or moderate in severity and not related to the study medication. A total of 26 patients experienced serious adverse events, all of which were deemed to be unrelated to migalastat.

“In conclusion, long-term migalastat treatment maintained the initial reduction in LVMi observed during phase 3 studies and was generally well tolerated,” the authors stated.

Disclosure: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Feldt-Rasmussen, Giugliani R, Hughes DA, et al. Long-term treatment with migalastat 150 mg every other day is associated with sustained cardiac efficacy and is well tolerated. Presented at: 17th Annual WORLDSymposium; February 8-12, 2020. Poster #070.