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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: 27 Mar 2026

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At a glance

  • Originator Genzyme Corporation
  • Developer Sanofi; Sanofi Genzyme
  • Class Antiparkinsonians; Azabicyclo compounds; Carbamates; 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

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

Most Recent Events

  • 18 Mar 2026 Sanofi announces intention to submit global regulatory application for venglustat for Gaucher's disease type III, in 2026
  • 18 Mar 2026 Venglustat - Sanofi receives Breakthrough Therapy status for Gaucher's disease type III in USA
  • 18 Mar 2026 Venglustat - Sanofi receives Orphan Drug status for Gaucher's disease type III in European Union, prior to March 2026

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