Sanofi’s Xenpozyme (olipudase alfa-rpcp) Receives the US FDA’s Approval as First Disease-Specific Treatment for Acid Sphingomyelinase Deficiency
Shots:
- The approval was based on the (ASCEND) & (ASCEND-Peds) trials that evaluated Xenpozyme in 31 adult & 8 pediatric patients with ASMD (non-CNS manifestations) type A/B or type B for 52 & 64wks. Xenpozyme is expected to be available in the US in the coming wks.
- The results showed a mean reduction in spleen volume by (38.9%) from baseline to 52wk. over mean increase by 0.5% for patients in the PBO group in (ASCEND) trial & 46.7% in (ASCEND-Peds) trials; mean reduction in liver volume (26.5% vs 1.8%) & 38.1%; mean improvement in platelet count (18.3% vs 2.7%) & 37.6%
- In (ASCEND-Peds) trials, patients who performed the test at baseline had a mean relative improvement of 45.9% predicted DLco from baseline (48.5%) to 52wk. (70.9%) along with an improvement in lung function in both trials
Ref: GlobeNewswire | Image: Sanofi
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