Sanofi (SNY.US) global first new drug application for listing.

On May 28, Sanofi (SNY.US) announced that the marketing application for Venglustat had been accepted by the FDA for priority review, for the treatment of type III Gaucher disease (GD3). If approved, Venglustat will become the first treatment in the United States for GD3-related progressive neurological manifestations, expanding Sanofi's treatment options for patients with lysosomal storage diseases. Venglustat is a novel oral glucosylceramide synthase inhibitor (GCSi) that can effectively inhibit N-terminal methyltransferase 1 (NTMT1) and can cross the blood-brain barrier. It has the potential to delay the progression of related diseases by inhibiting the accumulation of abnormal glycosphingolipids (GSLs) and their physiological consequences. Abnormal accumulation of GSLs can lead to impaired cellular function. The FDA's acceptance this time is mainly based on the positive results of the Phase III LEAP2MONO study. In this study, at week 52 of treatment, GD3 patients in the Venglustat group showed significant improvement in their Scale for the Assessment and Rating of Ataxia (SARA) total score and Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) score compared to the group receiving enzyme replacement therapy (imiglucerase) (p=0.007), indicating significant improvement in patient neurological symptoms. Furthermore, in the four key secondary endpoints reflecting non-neurological outcomes, the study met three of them, with significant improvements in spleen volume, liver volume, and hemoglobin levels in the Venglustat group. Gaucher disease (GD) is a rare genetic lysosomal storage disease caused by a deficiency of glucocerebrosidase (also known as acid -glucosidase), leading to accumulation of GSLs, especially in macrophages in the spleen, liver, bone marrow, and lungs.