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Date	Title and Summary	Additional Formats
Toggle SummaryMar 2, 2011	Positive Phase 2 Interim Data from First Study of Telaprevir in People Co-Infected with Hepatitis C and HIV Presented at CROI Conference Early results from ongoing study showed that the hepatitis C virus was undetectable by week 4 in 70% of people treated with telaprevir-based combination therapy HCV	View HTML Positive Phase 2 Interim Data from First Study of Telaprevir in People Co-Infected with Hepatitis C and HIV Presented at CROI Conference 131.7 KB
Toggle SummaryFeb 14, 2019	Positive Phase 3 Study for Tezacaftor/Ivacaftor Combination in Children Aged 6-11 Years with Cystic Fibrosis Supports European Medicines Agency Submission -Study met primary endpoint with a statistically significant improvement in absolute change in lung clearance index (LCI 2.5 ) through 8 weeks of tezacaftor/ivacaftor treatment- -Tezacaftor in combination with ivacaftor was generally well tolerated and safety data were consistent with previous General Business	View HTML PDF Version
Toggle SummaryNov 7, 2016	Positive Phase 3 Study of ORKAMBI® in Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies of the F508del Mutation Supports a Submission to the European Medicines Agency in the First Half of 2017 - Study met primary endpoint with a statistically significant improvement in absolute change in lung clearance index (LCI 2.5 ) compared to placebo through 24 weeks of treatment - - ORKAMBI was well tolerated with safety data that were similar to data from previous Phase 3 open-label safety study - Cystic Fibrosis	View HTML Positive Phase 3 Study of ORKAMBI® in Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies of the F508del Mutation Supports a Submission to the European Medicines Agency in the First Half of 2017 90.8 KB
Toggle SummaryJul 20, 2020	Positive Phase 3 Study Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older With Cystic Fibrosis Who Have One Copy of the F508del Mutation and One Gating or Residual Function Mutation -Phase 3 study met primary endpoint and all secondary endpoints- -Study is a U.S. post-marketing commitment and will be submitted to FDA- -Data also will be submitted to the European Medicines Agency to support indication expansion of the EU label following triple combination approval- BOSTON General Business	View HTML PDF Version
Toggle SummaryDec 11, 2023	Positive Results from Pivotal Trials of CASGEVY™ (exagamglogene autotemcel) Highlighted in Oral Presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition - Longer-term follow-up data demonstrate consistent and durable response to treatment - BOSTON --(BUSINESS WIRE)--Dec. 11, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from	View HTML PDF Version
Toggle SummaryJun 9, 2023	Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress - Both trials met the primary and key secondary endpoints at the pre-specified interim analysis - - Data continue to demonstrate transformative and durable benefit - - Safety profile consistent with busulfan conditioning and autologous hematopoietic stem cell transplant - BOSTON & ZUG,	View HTML PDF Version
Toggle SummaryOct 18, 2004	Preliminary Phase IIa Data for VX-702 Demonstrate Tolerability and Reduction in C-Reactive Protein in Cardiovascular Patients -First demonstration of CRP reduction in cardiovascular patients using an oral cytokine inhibitor- Other	View HTML Preliminary Phase IIa Data for VX-702 Demonstrate Tolerability and Reduction in C-Reactive Protein in Cardiovascular Patients 14.5 KB
Toggle SummaryJun 4, 2014	Proof-of-Concept Study of Ivacaftor Monotherapy Showed Improvements in Lung Function After Two Weeks of Treatment in People with Cystic Fibrosis who have a Residual Function Mutation -Data are consistent with in vitro observations in residual function mutations showing that ivacaftor improved CFTR activity- -8-week open-label period showed improvements in lung function- -Data support plans to initiate a Phase 3 study of ivacaftor in people with CF who have a residual function Cystic Fibrosis	View HTML Proof-of-Concept Study of Ivacaftor Monotherapy Showed Improvements in Lung Function After Two Weeks of Treatment in People with Cystic Fibrosis who have a Residual Function Mutation 35.1 KB
Toggle SummaryFeb 1, 2013	Republic of Ireland Approves Funding for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis, for People with a Specific Genetic Mutation (G551D) CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Health Service Executive (HSE) in the Republic of Ireland will fund KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), for people ages 6 and Cystic Fibrosis	View HTML Republic of Ireland Approves Funding for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis, for People with a Specific Genetic Mutation (G551D) 19.4 KB
Toggle SummaryApr 29, 2006	Researchers Report Initial Results for 14-day Phase Ib Study of VX-950, and Pegylated Interferon, Showing Anti-HCV Activity in Combination in Hepatitis C Patients -New data show that plasma HCV RNA levels were below limit of detection (10 IU/mL) in 8 of 8 patients continued on peg-IFN+RBV for 12 weeks- HCV	View HTML Researchers Report Initial Results for 14-day Phase Ib Study of VX-950, and Pegylated Interferon, Showing Anti-HCV Activity in Combination in Hepatitis C Patients 15.1 KB