Positive Results for olipudase alfa in two RCTs treamtent of acid sphingomyelinase deficiency (ASMD), or Nieman-Pick Disease in adults and children

In a phase 2/3 ASMD trial (n=36), olipudase alfa (a recombinant human acid sphingomyelinase) improved lung function by 22% from baseline at week 52 compared to 3% in the placebo arm; and reduced spleen volume by 39.5% compared to 0.5% in the placebo arm.

SPS commentary:

Olipudase alfa is also currently under investigation for this indication in phase 3 clinical trials, in the UK and EU.


Biospace Inc.