Pipeline

Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
  • Pain:
    VX-961

    • Phase 1
  • Alpha-1 Antitrypsin Deficiency:
    VX-814

    • Phase 1
  • APOL1-Mediated Kidney Diseases:
    VX-147

    • Phase 1
  • Alpha-1 Antitrypsin Deficiency:
    VX-864

    • Phase 1
  • Outlicensed:
    VX-803 (Outlicensed to Merck KGaA, Darmstadt, Germany)

    • Phase 1
  • Outlicensed:
    VX-984 (Outlicensed to Merck KGaA, Darmstadt, Germany)

    • Phase 1
  • Pain:
    VX-150

    • Phase 2
  • Outlicensed:
    VX-787 (Outlicensed to Janssen Pharmaceuticals, Inc.)

    • Phase 2
  • Outlicensed:
    VX-970 (Outlicensed to Merck KGaA, Darmstadt, Germany)

    • Phase 2
  • Cystic Fibrosis:
    VX-561

    • Phase 2
  • Cystic Fibrosis:
    VX-121 + TEZACAFTOR + VX-561

    • Phase 2
  • Cystic Fibrosis:
    VX-371 + LUMACAFTOR/IVACAFTOR

    • Phase 2
  • Muscular Dystrophy:
    Duchenne muscular dystrophy

    • Preclinical
  • Muscular Dystrophy:
    Myotonic dystrophy type 1

    • Preclinical
  • Hemoglobinopathies:
    BETA THALASSEMIA – CTX001

    • Phase 1/2
  • Hemoglobinopathies:
    SICKLE CELL DISEASE – CTX001

    • Phase 1/2
  • Cystic Fibrosis:
    Ivacaftor

    • Approved
  • Cystic Fibrosis:
    Lumacaftor/Ivacaftor

    • Approved
  • Cystic Fibrosis:
    Tezacaftor/Ivacaftor + Ivacaftor

    • Approved
  • Cystic Fibrosis:
    ELEXACAFTOR/TEZACAFTOR/IVACAFTOR + IVACAFTOR

    • Approved