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Date Title and Summary Additional Formats
Toggle Summary Vertex Receives CHMP Positive Opinion for KALYDECO® for the Treatment of Infants With Cystic Fibrosis Ages 1 Month and Older
- If approved, KALYDECO ® will be the first and only medicine approved in Europe to treat the underlying cause of cystic fibrosis in babies as young as 1 month with specific mutations in the CFTR gene - LONDON --(BUSINESS WIRE)--Feb. 23, 2024-- Vertex Pharmaceuticals (Nasdaq: VRTX) today announced
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Toggle Summary Vertex to Participate in Upcoming Investor Conferences
BOSTON --(BUSINESS WIRE)--Feb. 20, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Charles Wagner , Executive Vice President and Chief Financial Officer, and David Altshuler , M.D., Ph.D., Executive Vice President, Global Research , and Chief Scientific Officer, will
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Toggle Summary European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia may be eligible for treatment - LONDON --(BUSINESS WIRE)--Feb. 13, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has
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Toggle Summary Vertex Reports Fourth Quarter and Full Year 2023 Financial Results
— Full year product revenue of $9.87 billion , an 11% increase compared to full year 2022 — — Company provides full year 2024 product revenue guidance of $10.55 to $10.75 billion — — CASGEVY TM approved in the U.S. , Great Britain , the Kingdom of Saudi Arabia and Bahrain — — Vertex on track to
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Toggle Summary Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis
– Treatment with the once-daily vanza triple CFTR modulator regimen met all primary and key secondary endpoints in two randomized controlled trials in people with CF ages 12 years and older – – Results were more pronounced in the single-arm study in children ages 6 to 11 years, demonstrating the
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Toggle Summary Vertex Announces Positive Results From the VX-548 Phase 3 Program for the Treatment of Moderate-to-Severe Acute Pain
– Treatment with VX-548 led to statistically significant improvement in pain compared to placebo as well as a clinically meaningful reduction in pain from baseline in both the abdominoplasty and bunionectomy randomized controlled trials – – Treatment with VX-548 was also shown to be effective in
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Toggle Summary Vertex to Announce Fourth Quarter and Full Year 2023 Financial Results on February 5
BOSTON --(BUSINESS WIRE)--Jan. 17, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2023 financial results on Monday, February 5, 2024 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia
- Approximately 1,000 patients in the U.S. 12 years of age and older are now eligible for this one-time treatment - BOSTON --(BUSINESS WIRE)--Jan. 16, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™
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Toggle Summary Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia
- CASGEVY™ is first medicine ever to be evaluated through the SFDA’s Breakthrough Medicines Program - - First treatment center in Saudi Arabia has been activated - BOSTON --(BUSINESS WIRE)--Jan. 9, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and
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Toggle Summary Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings
BOSTON --(BUSINESS WIRE)--Jan. 7, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 42 nd Annual J.P. Morgan Healthcare Conference on Monday, January
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Toggle Summary Vertex to Present at the 42nd Annual J.P. Morgan Healthcare Conference on January 8
BOSTON --(BUSINESS WIRE)--Dec. 18, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani , Chief Executive Officer and President, will present at the 42 nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m.
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Toggle Summary Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
  - If approved by the European Commission , patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be
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Toggle Summary Vertex Announces Positive Results From Phase 2 Study of VX-548 for the Treatment of Painful Diabetic Peripheral Neuropathy
– Treatment with the NaV1.8 inhibitor VX-548 led to statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the Numeric Pain Rating Scale (NPRS) – – VX-548 was generally well tolerated – – Vertex plans to advance VX-548 into pivotal
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Toggle Summary 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
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Toggle Summary Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease
– First-ever approval of a CRISPR-based gene-editing therapy in the U.S.   – – Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment – – Multiple authorized treatment centers activated – BOSTON & ZUG, Switzerland
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Toggle Summary Vertex Appoints Nancy Thornberry to its Board of Directors
BOSTON --(BUSINESS WIRE)--Dec. 5, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Nancy Thornberry has been appointed to its Board of Directors as an independent director. Ms. Thornberry has more than 35 years of experience in the pharmaceutical and biotech
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Toggle Summary Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO® in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations
  -Application to add ~200 non-F508del CFTR mutations to the KAFTRIO ® license- -If approved, ~2,800 people with cystic fibrosis in the European Union ages 2 and above could receive a medicine that treats the underlying cause of their disease for the first time- LONDON --(BUSINESS WIRE)--Nov.
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Toggle Summary European Commission Approves KAFTRIO® in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5
-More than 1,200 children are newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON --(BUSINESS WIRE)--Nov. 23, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion
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Toggle Summary Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
  - First regulatory authorization of a CRISPR-based gene-editing therapy in the world – - CASGEVY is indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom hematopoietic
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Toggle Summary Vertex Reports Third Quarter 2023 Financial Results
— Product revenue of $2.48 billion , a 6% increase compared to Q3 2022 — — Company raises full year 2023 product revenue guidance to approximately $9.85 billion — — U.S. FDA Advisory Committee meeting for exa-cel in SCD completed; PDUFA date for exa-cel in SCD is December 8, 2023 — — Pipeline
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Toggle Summary Vertex to Present New Data on Exagamglogene Autotemcel (exa-cel) at the American Society of Hematology (ASH) Annual Meeting and Exposition
- New data including longer duration follow-up from SCD and TDT pivotal trials accepted for oral presentation – - Five additional abstracts accepted for poster presentation - BOSTON --(BUSINESS WIRE)--Nov. 2, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that seven
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Toggle Summary Vertex to Present Data Highlighting Long-Term Benefits of TRIKAFTA® at the North American Cystic Fibrosis Conference
BOSTON --(BUSINESS WIRE)--Nov. 2, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), which can treat the underlying cause of cystic fibrosis (CF) in ~90% of people with the disease, will be presented at
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Toggle Summary Vertex to Present at Upcoming Investor Conferences
BOSTON --(BUSINESS WIRE)--Oct. 26, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences. Charles Wagner , Executive Vice President and Chief Financial Officer, and David Altshuler , M.D., Ph.D., Executive Vice
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Toggle Summary Vertex to Announce Third Quarter 2023 Financial Results on November 6
BOSTON --(BUSINESS WIRE)--Oct. 10, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its third quarter 2023 financial results on Monday, November 6, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex Appoints Michel Lagarde to its Board of Directors
BOSTON --(BUSINESS WIRE)--Oct. 5, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Michel Lagarde has been appointed to its Board of Directors as an independent director. Mr. Lagarde is a senior health care and business leader with global expertise, currently serving
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Toggle Summary Vertex Presents Positive, Updated VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the European Association for the Study of Diabetes 59th Annual Meeting
- All patients treated with VX-880 in Parts A and B have follow-up data beyond Day 90 and have demonstrated islet cell engraftment and glucose-responsive insulin production - - All patients showed improvement across all measures of glucose control, including decreases in HbA1c, increases in blood
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Toggle Summary Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5
-If approved, more than 1,200   children would be newly eligible for a medicine that could treat the underlying cause of their disease- BOSTON --(BUSINESS WIRE)--Sep. 15, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee
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Toggle Summary Vertex Researchers Awarded 2024 Breakthrough Prize in Life Sciences
- Paul Negulescu , Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis – BOSTON --(BUSINESS WIRE)--Sep. 14, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that three of its researchers have
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Toggle Summary Vertex to Participate in the Morgan Stanley 21st Annual Global Healthcare Conference on September 13
BOSTON --(BUSINESS WIRE)--Aug. 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Morgan Stanley 21 st Annual Global Healthcare Conference. Dr. Reshma Kewalramani , Chief Executive Officer and President, and Charles Wagner , Executive Vice
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Toggle Summary Vertex Appoints E. Morrey Atkinson, Ph.D., to EVP and Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations
BOSTON --(BUSINESS WIRE)--Aug. 29, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that E. Morrey Atkinson , Ph.D., has been appointed Executive Vice President, Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations, effective
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Toggle Summary Vertex Announces Publication of VX-548 Positive Phase 2, Proof-of-Concept Results in New England Journal of Medicine
- First published manuscript of VX-548 preclinical and clinical trial results in a peer-reviewed journal - BOSTON --(BUSINESS WIRE)--Aug. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine (NEJM) of preclinical data and
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Toggle Summary Vertex Reports Second Quarter 2023 Financial Results
— Product revenue of $2.49 billion , a 14% increase compared to Q2 2022 — — Company raises full year 2023 product revenue guidance to $9.7 to $9.8 billion — — FDA has accepted the exa-cel BLAs in both severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT); Priority Review
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Toggle Summary European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old
-Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease- LONDON --(BUSINESS WIRE)--Jul. 5, 2023-- Vertex Pharmaceuticals today announced that the European Commission has
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Toggle Summary Vertex to Announce Second Quarter 2023 Financial Results on August 1
BOSTON --(BUSINESS WIRE)--Jun. 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second quarter 2023 financial results on Tuesday, August 1, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex and Lonza to Build Dedicated Manufacturing Facility for Type 1 Diabetes (T1D) Cell Therapies
- Following positive Phase 1/2 results in Vertex’s VX-880 program, Vertex and Lonza announce strategic manufacturing collaboration - - Dedicated large-scale facility will support commercial production of Vertex’s T1D cell therapy portfolio - - Approximately 300 new jobs anticipated at Lonza
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Toggle Summary Vertex Presents Positive VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the American Diabetes Association 83rd Scientific Sessions
–   All six patients treated with VX-880 engrafted islet cells, produced endogenous insulin (C-peptide) and had improved glycemic control while reducing or eliminating insulin use – –   The two patients with at least one year of follow-up met the criteria for the primary endpoint of elimination of
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Toggle Summary Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
-Interim results of largest real-world study of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed sustained improvement in lung function, reduction in pulmonary exacerbations frequency and lower rates of lung transplant and death for people with cystic fibrosis- - Twelve
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Toggle Summary 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 &amp; ZUG,
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Toggle Summary FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- First CRISPR gene-editing filings to be accepted for review by FDA - - FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) – - PDUFA target action date of December 8, 2023 , for SCD and March 30, 2024 , for TDT –
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Toggle Summary Vertex to Participate in the Goldman Sachs 44th Annual Global Healthcare Conference on June 13
BOSTON --(BUSINESS WIRE)--May 30, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Goldman Sachs 44 th Annual Global Healthcare Conference. Dr. Reshma Kewalramani , Chief Executive Officer and President, and Stuart A.
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Toggle Summary Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older
-First and only CFTR modulator approved for this age group- BOSTON --(BUSINESS WIRE)--May 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO ® (ivacaftor) for use in children with cystic fibrosis (CF) ages 1
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Toggle Summary Vertex Reports First Quarter 2023 Financial Results
— Product revenue of $2.37 billion , a 13% increase compared to Q1 2022 — — Company reiterates full year 2023 financial guidance, including product revenue guidance of $9.55 to $9.7 billion — — TRIKAFTA approved in U.S. for children with cystic fibrosis 2 to 5 years of age — — Pipeline advancement
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Toggle Summary Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old
- If approved by the European Commission, nearly 300 children with cystic fibrosis and two copies of the F508del mutation would be eligible for the first time for a medicine&nbsp;that can treat the underlying cause of their disease - LONDON – 26/27/28 April 2023 – Vertex Pharmaceuticals (Nasdaq: VRTX)
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Toggle Summary Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations
-About 900 children with cystic fibrosis will now have a medicine to treat the underlying cause of their disease for the first time- BOSTON --(BUSINESS WIRE)--Apr. 26, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved the
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Toggle Summary Vertex to Present at the Bank of America Securities 2023 Health Care Conference on May 9
BOSTON --(BUSINESS WIRE)--Apr. 25, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the Bank of America Securities 2023 Health Care Conference on Tuesday, May 9, 2023 at 9:20 a.m. PT / 12:20 p.m. ET .
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Toggle Summary Vertex to Announce First Quarter 2023 Financial Results on May 1
BOSTON --(BUSINESS WIRE)--Apr. 10, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2023 financial results on Monday, May 1, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
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Toggle Summary Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023 - BOSTON &amp; ZUG, Switzerland --(BUSINESS WIRE)--Apr. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced
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Toggle Summary Vertex and CRISPR Therapeutics Announce Licensing Agreement to Accelerate Development of Vertex’s Hypoimmune Cell Therapies for the Treatment of Type 1 Diabetes
- Vertex to receive non-exclusive rights to CRISPR Therapeutics’ CRISPR/Cas9 to accelerate development of potentially curative cell therapies for T1D- - CRISPR Therapeutics to receive $100M upfront payment plus milestone and royalty payments on potential future gene-edited hypoimmune T1D products-
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Toggle Summary Vertex Announces Publication in New England Journal of Medicine of Results from Phase 2 Study of Inaxaplin (VX-147)
BOSTON --(BUSINESS WIRE)--Mar. 16, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine   (NEJM)   of results from preclinical studies and a Phase 2 study evaluating the efficacy and safety of inaxaplin (VX-147) on top of
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Toggle Summary Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes
  – Vertex to initiate a Phase 1/2 clinical trial in the coming months – – VX-264 is the second investigational program in Vertex’s pipeline containing stem cell-derived, fully differentiated pancreatic islet cells for the treatment of type 1 diabetes – – VX-264 program does not require
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