Press Releases

- ALYFTREK ™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies - - In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV 1 and further decreased sweat chloride compared

- Approximately 300 more people with cystic fibrosis in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time - BOSTON --(BUSINESS WIRE)--Dec. 20, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S.

– Treatment with the highly selective NaV1.8 pain signal inhibitor suzetrigine met the primary endpoint with a statistically significant and clinically meaningful 2.02 point within-group reduction from baseline in the Numeric Pain Rating Scale (NPRS) – – Placebo arm showed similar within-group

- Data from long-term follow-up of patients in clinical trials further demonstrate durability of the transformative benefits of CASGEVY™ - - Safety profile consistent with busulfan conditioning and   autologous hematopoietic stem cell transplant - - Vertex provides update on progress in bringing

— Product revenue of $2.77 billion , a 12% increase compared to Q3 2023 — — Raising full-year product revenue guidance to $10.8 billion to $10.9 billion — — Preparing for two potential near-term launches: vanzacaftor triple in CF and suzetrigine (VX-548) for moderate-to-severe acute pain — —

BOSTON --(BUSINESS WIRE)--Oct. 29, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences. Dr. Reshma Kewalramani , Chief Executive Officer and President, will participate in a fireside chat at the Guggenheim Inaugural

- New data on povetacicept 80 mg SC Q4 weeks in IgA nephropathy shows mean UPCR reduction from baseline of 66% observed at 48 weeks, associated with stable renal function (eGFR) and 63% achieving clinical remission -- - First proteinuria data on povetacicept in primary membranous nephropathy shows

-- Phase 3 abstract selected for presentation in “Best Abstract” session -- BOSTON --(BUSINESS WIRE)--Oct. 18, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will present its pivotal Phase 3 data on suzetrigine, an investigational, oral, highly selective

BOSTON --(BUSINESS WIRE)--Oct. 10, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its third quarter 2024 financial results on Monday, November 4, 2024 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .

–   Phase 3 data on investigational vanza triple demonstrates non-inferiority to TRIKAFTA ® in ppFEV 1 and further improvement of CFTR function as measured by sweat chloride – –   Real-world evidence and clinical studies of TRIKAFTA ® continue to show sustained long-term benefits including

BOSTON --(BUSINESS WIRE)--Aug. 22, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences. Dr. Reshma Kewalramani , Chief Executive Officer and President, and Charles Wagner , Executive Vice President and Chief

- Eligible transfusion-dependent beta thalassemia (TDT) patients in England will be able to access the therapy from today - - CASGEVY is one of the first medicines funded by NHS England’s Innovative Medicines Fund - LONDON --(BUSINESS WIRE)--Aug. 7, 2024-- Vertex Pharmaceuticals (Nasdaq: VRTX)

— Product revenue of $2.65 billion , a 6% increase compared to Q2 2023 — — Company raises full year product revenue guidance to $10.65 to $10.85 billion — — FDA accepted NDA for vanzacaftor triple in CF with Priority Review and PDUFA target action date of January 2, 2025 ; additionally, MAA

– FDA grants priority review and assigns a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2025 – – Suzetrigine, an investigational non-opioid pain signal inhibitor, has the potential to treat millions of patients who suffer from moderate-to-severe acute pain each year –

- Vanza triple granted priority review with Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025 – - EU Marketing Authorization Application (MAA) submission also validated by European Medicines Agency (EMA) BOSTON --(BUSINESS WIRE)--Jul.

BOSTON --(BUSINESS WIRE)--Jul. 1, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second quarter 2024 financial results on Thursday, August 1, 2024 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .

– All 12 patients who received the full dose of VX-880 as a single infusion demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90 – – All patients achieved ADA -recommended target HbA1c levels 70% time-in-range (70-180 mg/dL), and 11 of 12 patients

- Results from CLIMB-111, -121 and -131 accepted for oral presentation - - Data from these trials, with the longest follow-up of more than five years, demonstrate transformative, consistent and durable benefit of CASGEVY™ - - Safety profile consistent with busulfan conditioning and   autologous

- Results from a randomized, placebo-controlled study of TRIKAFTA ® in people with cystic fibrosis with rare, non- F508del CFTR mutations showed statistically significant and clinically meaningful improvements in the primary and all secondary endpoints - - Interim results of largest real-world

BOSTON --(BUSINESS WIRE)--May 22, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences. Dr. Reshma Kewalramani , Chief Executive Officer and President, will present at the William Blair 44 th Annual Growth Stock