Sanofi announced mixed results for its rare disease drug venglustat in two Phase III clinical trials. The company reported that venglustat, a glucosylceramide synthase inhibitor, demonstrated neurological improvements in patients with Gaucher disease who have neurological symptoms. Gaucher disease is a rare genetic disorder that causes fatty substances to accumulate in tissue.
In the LEAP2MONO trial, venglustat was found to be more effective than enzyme replacement therapy (ERT), which is currently the standard treatment, in improving neurological symptoms at 52 weeks. Venglustat also matched ERT in other measures such as spleen and liver volume and hemoglobin levels. Based on these findings, Sanofi plans to seek regulatory approval for venglustat for use in Gaucher disease.
However, results from the PERIDOT trial for Fabry disease were less positive. The trial did not meet its primary goal of reducing neuropathic and abdominal pain in patients with Fabry disease, though there were reductions seen in related biomarkers. Sanofi stated it will continue to analyze these results and await further data from an ongoing Phase III study evaluating venglustat’s effect on left cardiac ventricular mass index in Fabry patients before making any additional regulatory decisions.
Sanofi has prior experience treating both diseases, manufacturing drugs like Fabrazyme for Fabry disease and Cerezyme and Cerdelga for Gaucher disease. Venglustat had previously received fast track designation from the FDA for both indications. The development of venglustat has faced challenges before; Sanofi ended a Phase II study of the drug for GM2 gangliosidosis after negative outcomes in 2024, and discontinued a combined Phase II/III kidney disease trial in 2021.
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