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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 Apr 2025

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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 - 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

  • 26 Dec 2024 Genzyme terminates phase-III AMETHIST trials in GM2 gangliosidoses (In adolescents, In children, In adults) in US, Germany, Portugal, France, the UK, Argentina, Austria, Brazil, Czech Republic, Italy, Japan, Russia, Spain, Turkey due to the absence of positive trends on clinical endpoints (PO) (NCT04221451)
  • 19 Feb 2024 Sanofi announces intention to submit regulatory application for Gaucher's disease (type 3) in 2026 or later (Sanofi pipeline, February 2024)
  • 18 Apr 2022 Phase-III clinical trials in Gaucher's disease type III (In children, In adults, In the elderly) in USA (PO) (NCT05222906) (EudraCT2021-005402-10)

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