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Research programme: recombinant protein therapeutics - Pharming/Transgenic Rabbit Models

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Drug Profile

Research programme: recombinant protein therapeutics - Pharming/Transgenic Rabbit Models

Alternative Names: Enzyme replacement therapies - Pharming/Transgenic rabbit models; POMPE; Protein replacement therapies - Pharming/Transgenic Rabbit Models; recombinant human-alpha-galactosidase; recombinant human-alpha-glucosidase; rh-alpha-galactosidase; rh-alpha-glucosidase

Latest Information Update: 17 May 2023

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At a glance

  • Originator Transgenic Rabbit Models
  • Developer Pharming Group NV
  • Class Alpha-glucosidases; Enzymes; Galactosidases; Recombinant proteins
  • Mechanism of Action Alpha-galactosidase replacements; Alpha-glucosidase replacements
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Fabry's disease; Glycogen storage disease type II

Most Recent Events

  • 11 May 2023 Discontinued - Preclinical for Glycogen storage disease type II in Netherlands (Parenteral)
  • 11 May 2023 Discontinued for Fabry's disease in Netherlands (Parenteral) (Pharming Group NV pipeline, May 2023)
  • 28 Oct 2022 No recent reports of development identified for research development in Fabry's-disease in Netherlands (Parenteral)

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