Rare Disease Report

Phase 3 Trial of Agalsidase Beta in Pediatric Patients with Fabry Disease

FEBRUARY 20, 2017
Andrew Black
A Phase 3b clinical trial assessing agalsidase beta in lowering pediatric Fabry patient’s globotriaosylceramide [GL-3] levels in skin, plasma and urine showed encouraging results for treatment.
The trial was a Phase 3b randomized, open-label, parallel-group study assessing agalsidase beta in ERT-naive male Fabry patients aged 5-18 years old. Skin biopsies were obtained at ERT-baseline at years 1, 3 and 5, or at early withdrawal.
 Patients were administered at low dose regimens, i.e. 0.5 mg/kg every 2 weeks (0.5q2w, n=16) or 1.0 mg/kg every 4 weeks (1.0q4w, n=15) for 5 years.


13 out of 20 (65%) patients with a non-zero superficial skin capillary endothelium (SSCE) GL-3 score at baseline reached and maintained zero scores (7 on 0.5q2w, 6 on 1.0q4w).
Shifts from baseline non-zero to zero SSCE GL-3 scores were statistically significant at each time point in the overall study population and both treatment groups, except at year 5 in the 1.0q4w group.
No patient had severe SSCE GL-3 accumulation (score of 3) at any time point. All patients with IgG peak titer ≤6400 showed complete and sustained GL-3 clearance in SSCE.
Patients with the highest peak IgG titers tended to show less robust GL-3 clearance in SSCE.


About agalsidase beta

Agalsidase beta is a recombinant human α-galactosidase A enzyme with the same amino acid sequence as the native enzyme.


Ramaswami U, et al. A randomized, phase 3B, open-label, parallel-group study of agalsidase beta in treatment-naive male pediatric patients with Fabry disease without severe symptoms (FIELD study): GL-3 clearance from superficial skin capillary endothelium. Presented at 13th Annual WORLDSymposium; February 13-17, 2017; San Diego, CA.

About Fabry disease

Fabry disease is an X-linked lysosomal storage disorder that leads to excessive deposition of globotriaosylceramide (GL-3) throughout the body. Skin, eye, kidney, heart, brain, and peripheral nervous system are highly vulnerable.
The condition is caused by deficiency of the enzyme alpha-galactosidase A (alpha-Gal A) which degrades GL-3. The accumulation of GL-3 is believed to cause a variety of symptoms, including pain, kidney failure, and increased risk of heart attack and stroke.

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