VERTEX PHARMACEUTICALS INC / MA

VRTX | Nasdaq | CIK: 0000875320 | SIC: 2834 Pharmaceutical Preparations | active
Vertex Pharmaceuticals is a global biotechnology company focused on transformative medicines for serious diseases. It markets TRIKAFTA/KAFTRIO, ALYFTREK, SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO for cystic fibrosis; CASGEVY (gene-editing therapy) for sickle cell disease and transfusion-dependent beta thalassemia; and JOURNAVX (suzetrigine) for acute pain. The company also has a mid- and late-stage pipeline targeting IgA nephropathy, APOL1-mediated kidney disease, neuropathic pain, type 1 diabetes, primary membranous nephropathy, and other serious diseases.
cystic-fibrosissickle-cell-diseasetransfusion-dependent-beta-thalassemiaacute-painiga-nephropathyapol1-mediated-kidney-diseaseperipheral-neuropathic-paintype-1-diabetesprimary-membranous-nephropathyautosomal-dominant-polycystic-kidney-diseasemyotonic-dystrophy-type-1crispr/cas9-gene-editingcftr-modulationnav1.8-selective-inhibitionallogeneic-stem-cell-derived-islet-cell-therapy

Products

NameTypeDescription
ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor)platformOnce-daily triple combination CFTR modulator for cystic fibrosis, approved 2024 for ages 6 and older.
TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor)platformTriple combination CFTR modulator for cystic fibrosis, approved 2019 for ages 2 and older.
SYMDEKO/SYMKEVI (tezacaftor/ivacaftor)platformDual combination CFTR modulator for cystic fibrosis, approved 2018 for ages 6 and older.
ORKAMBI (lumacaftor/ivacaftor)platformDual combination CFTR modulator for cystic fibrosis, approved 2015 for ages 1 and older.
KALYDECO (ivacaftor)platformCFTR modulator for cystic fibrosis, approved 2012 for ages 1 month and older.
CASGEVY (exagamglogene autotemcel)platformEx-vivo, non-viral CRISPR/Cas9 gene-editing therapy for severe SCD and TDT, approved 2023 for ages 12 and older.
JOURNAVX (suzetrigine)platformFirst-in-class oral selective NaV1.8 inhibitor for moderate-to-severe acute pain in adults, approved January 2025.

SEC Filings

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