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Venglustat - Sanofi

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Drug Profile

Venglustat - Sanofi

Alternative Names: Genz-682452-AA; Genz-682452-AU; GZ-402671; GZ/SAR402671; GZ/SAR402671A; Ibiglustat; SAR-402671; SAR402671A; Venglustat malate; Venglustat malate - Sanofi

Latest Information Update: 24 Sep 2026

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At a glance

  • Originator Genzyme Corporation
  • Developer Sanofi
  • Class Antiparkinsonians; Aza compounds; Azabicyclo compounds; Carbamates; Fluorocarbons; Heterocyclic bicyclo compounds; Small molecules; Thiazoles; Urologics
  • Mechanism of Action Glucosylceramide synthase inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Gaucher's disease type III; Polycystic kidney disease
  • New Molecular Entity Yes

Highest Development Phases

  • Preregistration Gaucher's disease type III
  • Phase II Gaucher's disease
  • Discontinued Fabry's disease; GM2 gangliosidoses; Parkinson's disease; Polycystic kidney disease

Most Recent Events

  • 08 Sep 2026 Sanofi plans a phase III trial for Gaucher's disease type III (In children, Treatment-experienced) in France, Germany, Hungary, Italy, Spain, Finland (PO, Tablet) in September 2026 (2026-525374-19-00)
  • 14 Aug 2026 Discontinued - Phase-II for Fabry's disease (Treatment-naive) in Czech Republic, France, United Kingdom, Poland, Russia, USA (PO) (Sanofi pipeline, August 2026)
  • 14 Aug 2026 Discontinued - Phase-II/III for Polycystic kidney disease in Italy, France, Denmark, China, Czech Republic, Canada, Australia, Belgium, Argentina, Portugal, Austria, USA, Turkey, Germany, Poland, Israel, South Korea, Taiwan, Romania, Netherlands, United Kingdom, Spain (PO) (Sanofi pipeline, August 2026)

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