On May 28, Sanofi announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for the New Drug Application (NDA) of Venglustat, intended for the treatment of Type 3 Gaucher Disease (GD3). If approved, Venglustat would become the first treatment in the United States for GD3-related progressive neurological manifestations and would expand Sanofi's portfolio of therapies for patients with lysosomal storage disorders.

Venglustat is a novel, oral, potent inhibitor of glucosylceramide synthase (GCSi). It strongly inhibits N-terminal methyltransferase 1 (NTMT1) and can cross the blood-brain barrier. It has the potential to slow disease progression by inhibiting the abnormal accumulation of glycosphingolipids (GSLs) and their pathophysiological consequences. Abnormal accumulation of GSLs leads to cellular dysfunction.

The FDA's acceptance is primarily based on positive results from the Phase III LEAP2MONO study. In this study, at Week 52, GD3 patients in the Venglustat group showed statistically significant improvement compared to the enzyme replacement therapy (imiglucerase) group in both the total score of the Scale for the Assessment and Rating of Ataxia (SARA) and the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) score (p=0.007), indicating significant improvement in neurological symptoms. Furthermore, the study met three out of four key secondary endpoints related to non-neurological outcomes, demonstrating significant improvements in the Venglustat group for spleen volume, liver volume, and hemoglobin levels.

Gaucher Disease (GD) is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme glucocerebrosidase (also known as acid β-glucosidase). This deficiency leads to the accumulation of GSLs, particularly in macrophages of the spleen, liver, bone marrow, and lungs.