Sanofi (SNY) announced that its investigational drug Venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease while it did not meet the primary endpoint in a phase 3 study of Fabry disease.
Venglustat is an investigational oral glucosylceramide synthase inhibitor (GCSi) designed to cross the blood-brain barrier and has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipid (GSL)accumulation and its pathophysiological consequences.
In the phase 3 study of type 3 Gaucher disease in adults and pediatric patients, dubbed LEAP2MONO, patients receiving Venglustat demonstrated statistically significant improvements in the primary endpoints of neurological symptoms measured by a global test score for two assessments, the Scale for Assessment and Rating of Ataxia (SARA) modified total score and the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), at week 52 compared with those receiving enzyme replacement therapy.
Venglustat also met three out of four key secondary endpoints of the trial, working as well as enzyme replacement therapy (ERT) on non-neurological outcomes, including changes in spleen volume, liver volume, and hemoglobin levels, three key secondary endpoints of the study.
Gaucher disease (GD) is a rare inherited lysosomal storage disease caused by a deficiency of the enzyme glucocerebrosidase leading to the accumulation of glycosphingolipids (GSLs), particularly in macrophages in the spleen, liver, bone marrow, and lungs.
Commenting on the LEAP2MONO study results, Houman Ashrafian, Executive Vice President and Head of Research and Development at Sanofi, said, "What excites us most is the potential to address critical unmet medical needs. A daily pill could make a big difference for Gaucher patients facing neurological challenges."
Sanofi plans to pursue global regulatory filings for Venglustat in type 3 Gaucher disease.
In the phase 3 study in Fabry disease, dubbed PERIDOT, Venglustat did not demonstrate superiority on the primary endpoint as both the Venglustat group and the placebo group experienced reductions in neuropathic and abdominal pain. Additional data are being analysed, with more information expected to be shared at a future medical meeting.
A second phase 3 study evaluating the effect of Venglustat on left cardiac ventricular mass index in men and women with Fabry disease, dubbed CARAT, is underway.
Fabry disease is an inherited rare lysosomal storage disease that results from a deficiency of functional alpha-galactosidase A (a-Gal A), leading to a build-up of GSLs, causing progressive cellular accumulation and organ damage in the kidney, cardiovascular and cerebrovascular systems.
Venglustat was previously granted orphan designation in the EU, the USA, and Japan for the potential treatment of both GD3 and Fabry disease. The U.S. Food and Drug Administration also granted fast-track designation to Venglustat for its potential use in GD3 and Fabry disease.
Over the year, SNY traded between $44.62 and $60.12. The stock closed Friday's trading at $47.04, up 1.82%.
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