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Nizubaglustat - Azafaros

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Drug Profile

Nizubaglustat - Azafaros

Alternative Names: AZ-3102

Latest Information Update: 23 Jul 2025

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At a glance

  • Originator Amsterdam UMC; Leiden University
  • Developer Azafaros; Leiden University
  • Class Aza compounds; Ethers; Fluorinated hydrocarbons; Piperidines; Small molecules
  • Mechanism of Action GBA2 protein inhibitors; Glucosylceramide synthase inhibitors
  • Orphan Drug Status

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

    Yes - GM1 gangliosidosis; Niemann-Pick disease type C; Sandhoff disease; GM2 gangliosidoses; Tay-Sachs disease
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase III GM1 gangliosidosis; GM2 gangliosidoses; Niemann-Pick disease type C
  • Preclinical Sandhoff disease

Most Recent Events

  • 30 Jun 2025 Phase-III clinical trials in GM1-gangliosidosis (In children, In adolescents, In adults, In the elderly) in Portugal (PO) (NCT07054515)
  • 30 Jun 2025 Phase-III clinical trials in GM2 gangliosidoses (In adolescents, In children, In the elderly, In adults) in Portugal (PO) (NCT07054515)
  • 30 Jun 2025 Phase-III clinical trials in Niemann-Pick disease type C (In adolescents, In adults, In children, In the elderly) in Portugal (PO) (NCT07054515)

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