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Saturday, August 30, 2025

Amicus Therapeutics

 

Amicus Therapeutics products

A

  • Galafold (migalastat)
    Indication: Fabry disease in adults with an amenable GLA gene variant
    Dose: 123 mg orally once every other day, on an empty stomach

P

  • Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat)
    Indication: Late-onset Pompe disease in adults weighing ≥ 40 kg who are not improving on current enzyme replacement therapy
    Dose: Pombiliti 20 mg/kg IV infusion every other week over ~4 hours, combined with Opfolda 260 mg orally (or 195 mg if 40–<50 kg)

A

  • Amicus Assist
    Description: Patient support and assistance program (not a therapeutic product)


D – Developmental / Pipeline

  • SD-101 (Zorblisa)
    Indication: Epidermolysis Bullosa (rare blistering skin disorder)
    Status: Reached Phase 3 development, later discontinued

  • Afegostat (Plicera; isofagomine)
    Indication: Gaucher’s disease (pharmacological chaperone candidate)
    Status: Development terminated after Phase 2 trial



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