March 29, 2012
/PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, presented data that show favorable treatment effect and tolerability of REPLAGAL
(agalsidase alfa) in Fabry patients who switched from Fabrazyme
(agalsidase beta) or were naive to enzyme replacement therapy (ERT) after one year of treatment with REPLAGAL at the America College of Medical Genetics (ACMG) annual meeting in
Fabry disease is a rare, X-linked, lysosomal storage disorder caused by insufficient activity of the alpha-galactosidase enzyme. As a result of this deficiency, patients experience a spectrum of signs and symptoms including impairment in renal and cardiac functions. The natural course of Fabry disease includes decrease of renal function and increase in left ventricular mass index (LVMI).
"It's common for Fabry patients to suffer from chronic kidney and cardiovascular disease; these are signs of disease progression," said Dr.
, Director at Lysosomal Disorders Research and Treatment Unit, Center for Clinical Trials,
. "These interim results show the effectiveness and tolerability of REPLAGAL in Fabry patients after one year of treatment."
Data Suggest Favorable Treatment Effect and Tolerability with REPLAGAL at the Recommended Dose of 0.2 mg/kg Body Weight after One Year of Treatment
One year data from Shire's HGT-REP-059 multicenter, open-label treatment protocol, suggests that cardiac structure, as measured by LVMI, remained stable in treatment-naive and switch patients after one year of treatment with REPLAGAL. In treatment naive patients (n=22), mean LVMI was 48.5 g/m(2.7) at baseline and 50.7 g/m(2.7) at 12 months, an increase of 2.20 g/m(2.7) +/- 1.63; p=0.187. In switch patients (n=39), mean LVMI was 60.4 g/m(2.7) at baseline and remained virtually unchanged at 12 months at 60.3 g/m(2.7) (0.00 +/- 2.32; p=0.306). The small increases of LVMI observed in the study were not statistically significant and were below the progression expected from natural history data.