Press Releases

–   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

-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

- 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,

- 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 –

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.

-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

— 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

- 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 that can treat the underlying cause of their disease - LONDON – 26/27/28 April 2023 – Vertex Pharmaceuticals (Nasdaq: VRTX)

-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

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 .

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 .

-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023 - BOSTON & ZUG, Switzerland --(BUSINESS WIRE)--Apr. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced

- 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-

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

  – 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

BOSTON --(BUSINESS WIRE)--Feb. 21, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at Cowen’s 43 rd Annual Health Care Conference on Tuesday, March 7, 2023 at 2:50 p.m. ET . A live webcast of management's remarks will be available through the

— Full year product revenue of $8.93 billion , an 18% increase compared to full year 2021 — — Full year GAAP and non-GAAP net income increased 42% and 53%, respectively, versus 2021— — Company provides full year 2023 product revenue guidance of $9.55 to $9.7 billion — — Exa-cel regulatory

BOSTON --(BUSINESS WIRE)--Jan. 17, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2022 financial results on Tuesday, February 7, 2023 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .

BOSTON --(BUSINESS WIRE)--Jan. 4, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the 41 st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023 at 11:15 a.m. ET / 8:15 a.m. PT .

- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators - - VX-522 clinical trial in people with CF to initiate in coming weeks - BOSTON --(BUSINESS WIRE)--Dec. 12, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that