Vertex Pharmaceuticals Incorporated

Quick facts

Marketed products

• Immunosuppressant Regimen
• IVA · Pulmonary/Respiratory
  IVA is a CFTR potentiator that increases the open probability of the cystic fibrosis transmembrane conductance regulator protein to enhance chloride ion transport.
• LUM · Gastroenterology
  LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function.
• LUM/IVA · Respiratory / Genetic disease
  LUM/IVA is a combination of lumacaftor (a CFTR corrector) and ivacaftor (a CFTR potentiator) that work together to improve the processing and function of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein.
• Pegylated Interferon Alfa 2a · Immunology, Virology, Oncology
  Pegylated Interferon Alfa 2a activates innate immune responses by binding to interferon-alpha receptors, enhancing antiviral and antiproliferative activity.
• Ribavirin (Copegus®) · Virology/Infectious Disease
  Ribavirin is a nucleoside analog that inhibits viral RNA synthesis by interfering with guanosine triphosphate pools and viral polymerase activity.
• SUZ · Pulmonary/Respiratory
  SUZ is a CFTR potentiator that enhances the function of cystic fibrosis transmembrane conductance regulator protein to improve chloride transport in cystic fibrosis patients.
• Suzetrigine (SUZ) · Pain management / Neurology
  Suzetrigine is a selective sodium channel inhibitor that reduces neuronal excitability by blocking voltage-gated sodium channels.
• TEZ · Pulmonary/Genetic Disorders
  TEZ (tezacaftor) is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly and traffic to the cell surface.

Phase 3 pipeline

• CTX001 · Oncology / Hematologic Malignancies
  CTX001 is a genetically modified T-cell therapy that enhances patient immune cells to recognize and destroy cancer cells expressing specific antigens.
• ELX/TEZ/IVA · Respiratory / Genetic Disease
  ELX/TEZ/IVA is a triple combination of CFTR modulators that work together to restore function of the cystic fibrosis transmembrane conductance regulator protein.
• Exa-cel · Hematology/Rare Genetic Disorders
  Exa-cel is an in vivo CRISPR gene-editing therapy that modifies the BCL11A gene to reactivate fetal hemoglobin production in patients with sickle cell disease or beta-thalassemia.
• HB/APAP · Virology / Hepatology
  HB/APAP is a combination therapy that pairs a hepatitis B antigen with acetaminophen to modulate immune responses against hepatitis B infection.
• Placebo (matched to HB/APAP)
  A placebo control matched to the active comparator HB/APAP (hydrocodone bitartrate/acetaminophen) that contains no pharmacologically active ingredients.
• Placebo (matched to IVA)
  Placebo is an inert control substance with no active pharmacological mechanism.
• Placebo (matched to ivacaftor)
  This drug has no therapeutic effect.
• Placebo (matched to SUZ)
  Placebo is an inert substance with no active pharmacological mechanism.
• Placebo matched to VX-661/ ivacaftor · Pulmonary / Genetic Disorders
  This is a placebo control arm in a clinical trial comparing VX-661 (a CFTR corrector) combined with ivacaftor (a CFTR potentiator) against placebo in cystic fibrosis patients.
• TEZ/IVA · Pulmonology
  TEZ/IVA is a combination of tezacaftor and ivacaftor, which work together to increase the function of the CFTR protein in the lungs.
• Tezacaftor/Ivacaftor · Pulmonary/Respiratory
  Tezacaftor is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly, while ivacaftor is a CFTR potentiator that increases the channel-opening probability of CFTR at the cell surface.
• VNZ/TEZ/D-IVA · Cystic Fibrosis
  VNZ/TEZ/D-IVA is a triple combination therapy consisting of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, a corrector, and a potentiator.
• VX-121/TEZ/D-IVA · Pulmonology
  VX-121/TEZ/D-IVA is a triple combination of a cystic fibrosis transmembrane conductance regulator (CFTR) corrector (VX-121), a CFTR potentiator (tezacaftor), and a CFTR amplifier (ivacaftor).
• VX-445/TEZ/IVA · Pulmonology
  VX-445/TEZ/IVA is a combination of three cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
• VX-661/Ivacaftor · Respiratory
  VX-661/Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
• VX-661 plus ivacaftor combination · Respiratory
  VX-661 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, and ivacaftor is a CFTR potentiator.
• VX-770 · Respiratory / Genetic Disorders
  VX-770 is a CFTR potentiator that increases the opening probability and duration of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel at the cell surface.
• VX-809 · Cystic Fibrosis
  VX-809 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
• VX-880 · Ophthalmology
  VX-880 is a stem cell therapy that aims to restore vision in patients with advanced retinitis pigmentosa.

Phase 2 pipeline

• Ivacaftor Exposed · Respiratory
  Potentiates CFTR chloride channel function
• VX-147
• VX-150
• VX-152
• VX-407
• VX-440
• VX-509
• VX-548
• VX-659
• VX-661
• VX-670
• VX-765
• VX-787
• VX-950
• VX-993

Phase 1 pipeline

• 14C-VX-993
• AVN-944 capsules for oral administration
• Cethrin · Other
• telaprevir formulation A
• telaprevir Formulation B
• telaprevir Formulation C
• telaprevir Formulation D
• VCH-222
• VCH 916
• VX-118
• VX-264
• VX-272
• VX-445
• VX-522 mRNA therapy
• VX-581
• VX-634
• VX-668
• VX-708
• VX-809 & VX-770
• VX-828
• VX-840
• VX-973

Competitive intelligence

For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:

• Vertex Pharmaceuticals Incorporated portfolio CI brief
• Vertex Pharmaceuticals Incorporated pipeline updates RSS