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