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
Click here to read the full press release
Tags
Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
