Press Releases

- 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

BOSTON --(BUSINESS WIRE)--May 15, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Jennifer Schneider , M.D., M.S., has been elected to its Board of Directors as an independent director. Dr. Schneider has more than two decades of experience in the health care industry

— Product revenue of $2.69 billion , a 13% increase compared to Q1 2023; reiterated full year 2024 financial guidance, including product revenue guidance of $10.55 to $10.75 billion — — Submitted NDA and MAA filings for vanzacaftor triple in CF to FDA and EMA, respectively — — Initiated rolling NDA

-   KALYDECO   ® is the first and only medicine approved in the EU in this age group to treat the underlying cause of cystic fibrosis for specific mutations in the CFTR gene - LONDON --(BUSINESS WIRE)--Apr. 26, 2024-- Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European

Vertex obtains an exclusive license to TreeFrog's C-Stem TM   manufacturing technology in type 1 diabetes  TreeFrog and Vertex to collaborate on scale-up of fully differentiated, insulin-producing pancreatic islet cells  BOSTON and BORDEAUX, France , April 23, 2024 /PRNewswire/ -- Vertex

– Rolling submission for suzetrigine moderate-to-severe acute pain NDA granted by FDA; first module submitted and on track to complete filing this quarter – – Successful completion of end-of-phase 2 FDA meeting for pain associated with diabetic peripheral neuropathy (DPN); Phase 3 program to

- Alpine is a clinical stage biotechnology company focused on discovering and developing innovative, protein-based immunotherapies - - Alpine’s lead product, povetacicept, demonstrated best-in-class potential in patients with IgA nephropathy (IgAN); Phase 3 to initiate in H2 2024 - - Povetacicept

BOSTON --(BUSINESS WIRE)--Apr. 9, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2024 financial results on Monday, May 6, 2024 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .

– 45 mg once daily oral dose selected for Phase 3 – – Results support trial expansion to lower age group and study will now include adolescents ages 10-17 years – – If positive, pre-planned interim analysis at Week 48 may serve as the basis for accelerated approval in the U.S.

– ADPKD is the most common inherited kidney disease, with no treatments currently available that address the underlying cause of disease – – Vertex to initiate a Phase 1 clinical trial in healthy volunteers this month – – ADPKD is Vertex’s 10th disease area in the clinic, further expanding the

- 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

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

- 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

— 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

– 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