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Monday, August 25, 2025

Vertex Pharmaceuticals Incorporated


Vertex Pharmaceuticals – Product Catalog

  • Exa-cel (Casgevy™ in U.S./UK, Casgevy™/Exagamglogene autotemcel) → Generic: exagamglogene autotemcel (CRISPR/Cas9 edited autologous HSC therapy) → Indication: Sickle cell disease (SCD) with vaso-occlusive crises, transfusion-dependent beta thalassemia → Dose: One-time autologous infusion (gene-edited hematopoietic stem cells)

  • Kalydeco → Generic: ivacaftor → Indication: Cystic fibrosis with specific CFTR gating mutations → Dose: Tablets 150 mg bid (≥6 years); oral granules 50–75 mg for pediatrics

  • Orkambi → Generic: lumacaftor + ivacaftor → Indication: Cystic fibrosis in patients homozygous for F508del mutation → Dose: Tablets 200/125 mg; oral granules for ages ≥1 year

  • Symdeko / Symkevi (EU) → Generic: tezacaftor + ivacaftor (with or without ivacaftor boost) → Indication: Cystic fibrosis (specific CFTR mutations including F508del heterozygotes) → Dose: Tablets 100/150 mg + ivacaftor 150 mg

  • Trikafta / Kaftrio (EU) → Generic: elexacaftor + tezacaftor + ivacaftor (plus ivacaftor booster) → Indication: Cystic fibrosis with ≥1 F508del mutation (broadest CF coverage) → Dose: Combo tablets 100/50/75 mg (am dose) + ivacaftor 150 mg (pm dose)



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