Sunday, August 31, 2025
Saturday, August 30, 2025
Amicus Therapeutics
Amicus Therapeutics products
A
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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
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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
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Amicus Assist
Description: Patient support and assistance program (not a therapeutic product)
D – Developmental / Pipeline
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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