Shire Pharmaceuticals reported further results of its clinical trial evaluating its investigational human enzyme replacement therapy, idursulfase, for the treatment of Hunter syndrome. The data were presented at the annual meeting of the American Society of Human Genetics in Salt Lake City, Utah.
The primary efficacy endpoint of the trial was a composite of two clinical measures -- forced vital capacity and a six-minute walking test. As previously reported, patients receiving the weekly dosing regimen of 0.5 mg/kg of idursulfase showed a statistically significant difference compared to placebo. Patients receiving the every-other-week dosing regimen of idursulfase also showed a statistically significant difference compared to placebo when measuring the composite. Treatment with idursulfase was generally well-tolerated by patients in the trial.
Other results presented today included urine GAG levels, liver and spleen volume, cardiac left ventricular volume, and joint range of motion
