Olipudase alfa: Phase Ib data

An open-label, dose-escalation, U.S. and U.K. Phase Ib trial in 5 adults with non-neuronopathic Niemann-Pick type B disease showed that IV olipudase alfa every 2 weeks was well tolerated from an initial dose of 0.1 mg/kg to the maximum dose of 3 mg/kg. No serious or severe adverse events reported and no patients developed anti-olipudase alfa antibodies. The most common treatment-emergent adverse events reported were infusion site reactions, headache, arthralgia, abdominal pain and nausea. Olipudase alfa led to a 27.5 percentage point reduction and an 87% relative reduction in mean sphingomyelin content of total liver tissue from baseline at week 26. ...