Shots:
- The US FDA has accepted NDA & granted priority review to Sanofi‘s venglustat for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), with the PDUFA action date of Nov 25, 2026
- NDA was supported by the ongoing P-III (LEAP2MONO) trial assessing venglustat (QD, PO) vs enzyme replacement therapy (IV, Q2W) in 43 pts (≥12yrs.) with GD3, which met both of its 1EP & achieved 3 of 4 key 2EPs
- Venglustat is also under regulatory review for GD3 in the EU, with Sanofi planning to pursue additional global regulatory filings for venglustat in 2026
Ref: Sanofi | Image: Sanofi | Press Release
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