Vertex Pharmaceuticals Incorporated engages in the business of discovering, developing, manufacturing, and commercializing medicines for serious diseases. The company develops and commercializes therapies for the treatment of cystic fibrosis (CF), and advances its research and development programs in other indications. CF ORKAMBI ORKAMBI (lumacaftor in combination with ivacaftor) is approved by the United States Food and Drug Administration (FDA), and by the European Commission in 2015, for the treatment of patients with CF 12 years of age and older who have 2 copies (homozygous) of the F508del mutation in their CF transmembrane conductance regulator (CFTR) gene. The company completed the first of two Phase 3 clinical trials evaluating lumacaftor in combination with ivacaftor for the treatment of patients with CF 6 to 11 years of age who are homozygous for the F508del mutation in their CFTR gene. The company has submitted regulatory applications seeking approval for lumacaftor in combination with ivacaftor in Australia based on TRAFFIC and TRANSPORT, two Phase 3 randomized, double-blind, placebo-controlled clinical trials of lumacaftor in combination with ivacaftor that it completed in 2014. The company engages in various other research and mid- and early-stage development programs, including programs in the areas of oncology, pain and neurology. KALYDECO KALYDECO (ivacaftor) is an orally-administered CFTR potentiator that is approved in the United States, European Union, Australia and Canada for the treatment of certain patients with CF who have specific mutations in their CFTR gene. In the United States, KALYDECO is approved for the treatment of patients with CF two years of age and older who have one of the mutations in their CFTR gene, including G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and R117H. In the European Union, KALYDECO is approved for the treatment of patients with CF two years of age and older who have one of the following mutations in their CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D; and 18 years of age and older who have the R117H mutation in their CFTR gene. In October 2015, the company submitted a supplemental New Drug Application to the FDA for KALYDECO for patients with CF two years of age and older who have 1 of 23 residual function mutations. CF Development Programs The company has multiple development programs in the field of CF, including VX-661, a corrector compound that it is evaluating in a Phase 3 development program in combination with ivacaftor in multiple CF patient populations who have at least one copy of the F508del mutation in their CFTR gene; VX-371, an investigational epithelial sodium channel (ENaC), inhibitor, that is being evaluated in a Phase 2 development program and which it exclusively licensed from Parion Sciences, Inc. (Parion), in 2015; and VX-152 and VX-440, two next-generation CFTR corrector compounds that entered Phase 1 clinical trials in the fourth quarter of 2015 and that it plans to evaluate as part of combination treatment regimens. VX-661 in Combination with Ivacaftor VX-661 is an orally-administered CFTR corrector drug candidate that the company is developing in combination with ivacaftor. In 2015, the company initiated a Phase 3 development program comprised of four separate clinical trials for VX-661 in combination with ivacaftor in multiple CF patient populations who have at least one copy of the F508del mutation in their CFTR gene. ENaC Inhibition In 2015, the company entered into a collaboration with Parion to develop investigational ENaC inhibitors, including VX-371, for the potential treatment of CF and other pulmonary diseases. VX-371 is being evaluated in an exploratory Phase 2a clinical trial in approximately 120 patients with CF with any mutation in their CFTR gene, including those who have mutations not expected to respond to ivacaftor alone. Next-generation CFTR Corrector Compounds The company is developing two next-generation CFTR corrector compounds, VX-152 and VX-440, that it plans to evaluate as part of triple combination treatment regime
vertex pharmaceuticals inc
50 Northern Avenue
Boston, MA 02210
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