Venglustat

Unassigned

New Medicines

Autosomal dominant polycystic kidney disease (ADPKD)

Information

New molecular entity
Sanofi Genzyme
Sanofi Genzyme

Development and Regulatory status

None
None
Phase III Clinical Trials

Category

Glucosylceramide synthase inhibitor
Autosomal dominant polycystic kidney disease affects as many as 1 in 1,000 individuals. It accounts for about 10% of people on dialysis [1].
Autosomal dominant polycystic kidney disease (ADPKD)
Oral

Fabry's disease

Information

Licence extension / variation
Sanofi Genzyme
Sanofi Genzyme

Development and Regulatory status

None
Phase II Clinical Trials
Phase II Clinical Trials

Category

A glucosylceramide synthase inhibitor that works by blocking the formation of glucosylceramide (GL-1), a key component in the production of GL-3.
Fabry disease, which affects around 10,000 people around the world, is characterised by excessive accumulation of a fatty substance - glucosylceramide, also known as GL-3 or Gb3 - throughout the body, potentially leading to life threatening renal, cardiac and cerebrovascular events [1].
Fabry's disease
Oral