Sanofi Initiates Phase 3 Clinical Trial of Venglustat for Fabry Disease in China

Recently, the official website of the Chinese Drug Clinical Trial Registration and Information Disclosure Platform announced that Sanofi has launched an international multicenter Phase 3 clinical study in China for venglustat in patients with Fabry disease. Venglustat is an oral GCS inhibitor, and Sanofi is currently conducting Phase 2/3 clinical trials of this drug for multiple rare disease indications.

Screenshot source: China Drug Clinical Trial Registration and Information Disclosure Platform

Venglustat is an orally administered therapy capable of crossing the blood-brain barrier and acts as a glucosylceramide synthase (GCS) inhibitor. GCS is a key regulator in the glycosphingolipid (GSL) metabolic pathway, and by inhibiting GCS activity, venglustat can treat various rare diseases caused by genetic mutations in the glycosphingolipid metabolic pathway, such as Fabry disease.

Previously, the results of a 3-year Phase 2 clinical study conducted on patients with Fabry disease showed that it could reduce the accumulation of GL-3 in patients' cells, demonstrating preliminary efficacy. Public data indicates that reducing the accumulation of GL-3 in the cells of Fabry disease patients can alleviate acroparesthesia and gastrointestinal symptoms, improve myocardial hypertrophy, stabilize renal function, and thereby enhance the patients' quality of life and prognosis.

According to information on Sanofi's official website, venglustat is currently undergoing phase 2/3 clinical trials overseas for indications such as GM2 gangliosidosis, type III Gaucher disease (GD3), Fabry disease, and autosomal dominant polycystic kidney disease. In China, the drug has also received multiple implied clinical trial approvals, with proposed indications including Fabry disease, autosomal dominant polycystic kidney disease, and type III Gaucher disease.

This time, Sanofi has registered a randomized, open-label, parallel-group, 18-month international multicenter Phase 3 study. The primary objective is to explore the effect of venglustat treatment over 18 months on the left ventricular mass index in Fabry disease patients with left ventricular hypertrophy, compared to standard treatment for Fabry disease. The principal investigator in China is Dr. Wei Jin from Ruijin Hospital, affiliated with Shanghai Jiao Tong University School of Medicine. The study plans to enroll 12 participants in China and 90 participants internationally.

Fabry disease is an X-linked genetic disorder caused by mutations in the GLA gene, which encodes α-galactosidase A (α-Gal A) on the long arm of the X chromosome. These mutations result in structural and functional abnormalities of α-Gal A, leading to the accumulation of its metabolic substrates, globotriaosylceramide (GL-3), and related glycosphingolipids in multiple organs throughout the body, thereby causing a clinical syndrome. The disease manifests as multi-organ and multi-system involvement, with different organs being affected progressively with age, such as ocular involvement leading to reduced vision or even blindness, and neurological involvement causing unbearable burning sensations in the soles of the feet and palms.

▽Follow 【WuXi AppTec】Germany】WeChat Official Account