Alpha galactosidase: Interim Phase I/II data

Interim data from 12 evaluable treatment-naïve patients with Fabry’s disease in an open-label, dose-escalation, international Phase I/II trial showed that 0.2 and 1 mg/kg doses of IV PRX-102 given every 2 weeks were well tolerated. There were no changes in cardiac and kidney functions from baseline to month 6 as measured by MRI. The most common treatment-related adverse events reported were partial right conjunctival edema, chest tightening, nausea, subconjuctival hemorrhage, loose bowels, rash, lightheadedness, asymptomatic hypotension, vomiting, pain in the hands, tightness in the lower legs, shortness of breath and facial flushing. There was 1 discontinuation due to a grade 3 bronchospasm. ...