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Research programme: enzyme replacement therapy - Chiesi Farmaceutici

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Drug Profile

Research programme: enzyme replacement therapy - Chiesi Farmaceutici

Alternative Names: Agazym; Aspartylglucosaminuria; Galaczym; Recombinant lysosomal galactocerebrosidase; rhAGA; rhGALC; ZA 011

Latest Information Update: 27 Oct 2025

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At a glance

  • Originator Zymenex A/S
  • Developer Chiesi Farmaceutici
  • Class Enzymes; Recombinant proteins
  • Mechanism of Action Amidohydrolase replacements; Galactosylceramidase replacements
  • Orphan Drug Status

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

    Yes - Globoid cell leukodystrophy; Lysosomal storage diseases
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical CNS disorders; Unspecified
  • Discontinued Globoid cell leukodystrophy; Lysosomal storage diseases

Most Recent Events

  • 27 Oct 2025 Preclinical development is ongoing in CNS disorders in Italy (unspecified route)
  • 27 Oct 2025 Preclinical development is ongoing in Unspecified in Italy (unspecified route)
  • 28 Feb 2023 Discontinued - Preclinical for Globoid cell leukodystrophy in Denmark (unspecified route) (Chiesi pipeline, February 2023)

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