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Cipaglucosidase alfa - Amicus Therapeutics

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Drug Profile

Cipaglucosidase alfa - Amicus Therapeutics

Alternative Names: AT B200; cipaglucosidase alfa-atga; Pombiliti; Recombinant-human-acid-alpha-glucosidase-enzyme-Amicus; rhGAA-Amicus

Latest Information Update: 05 May 2026

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At a glance

  • Originator Amicus Therapeutics
  • Class Alpha-glucosidases; Enzymes
  • Mechanism of Action Alpha-glucosidase replacements
  • Orphan Drug Status

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

    Yes - Glycogen storage disease type II
  • New Molecular Entity No

Highest Development Phases

  • Marketed Glycogen storage disease type II

Most Recent Events

  • 27 Apr 2026 Amicus Therapeutics has been acquired by BioMarin Pharmaceutical
  • 25 Jun 2025 Preregistration for Glycogen storage disease type II (Combination therapy) in Japan (IV) prior to June 2025
  • 25 Jun 2025 Registered for Glycogen storage disease type II (Combination therapy) in Japan (IV)

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