Collaboration to leverage Orna's differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties WATERTOWN, Mass. , Jan. 7, 2025 /PRNewswire/ -- Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year... Read More

BOSTON / Dec 23, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 43 rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 a.m. ET/7:30 a.m. PT. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section... Read More

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 to TRIKAFTA ® BOSTON / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S.... Read More

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 / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of... Read More

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 reduction in NPRS Suzetrigine was generally well tolerated Advancement to Phase 3 in painful lumbosacral radiculopathy planned, pending... Read More

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 CASGEVY to patients BOSTON / Dec 08, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data... Read More

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 Pipeline progress continuing with three additional programs advancing to Phase 3: suzetrigine in DPN, povetacicept in IgAN and VX-880 in T1D BOSTON /... Read More

BOSTON / Oct 29, 2024 / Business Wire / 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 Healthcare Innovation Conference on Tuesday, November 12, 2024 at 9:00 a.m. ET. Stuart A. Arbuckle, Executive Vice President and... Read More

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 mean UPCR reduction from baseline of 62% at 24 weeks – - Global Phase 3 RAINIER trial of povetacicept in IgA nephropathy now underway... Read More

Phase 3 abstract selected for presentation in “Best Abstract” session BOSTON / Oct 18, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will present its pivotal Phase 3 data on suzetrigine, an investigational, oral, highly selective NaV1.8 pain signal inhibitor for the treatment of moderate-to-severe acute pain, at the annual meeting of the American Society of... Read More

BOSTON / Oct 10, 2024 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals Third Quarter 2024... Read More

TORONTO , Sept. 26, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has accepted for review its New Drug Submission (NDS) for vanzacaftor/tezacaftor/deutivacaftor, a once-daily triple combination therapy for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane... Read More

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 improvement in pancreatic function in young patients BOSTON / Sep 26, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)... Read More

First regulatory authorization of a CRISPR-based gene-editing therapy in Canada TORONTO , Sept. 25, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for Pr CASGEVY ® (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease (SCD)... Read More

BOSTON / Aug 22, 2024 / Business Wire / 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 Financial Officer, will participate in a fireside chat at the Morgan Stanley 22 nd Annual Global Healthcare Conference on Thursday,... Read More

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 / Aug 07, 2024 / Business Wire / Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible transfusion-dependent beta thalassemia (TDT) patients to access the... Read More

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 submissions validated in EU and U.K. FDA accepted NDA for suzetrigine (VX-548) for moderate-to-severe acute pain with Priority Review and PDUFA... Read More

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 BOSTON / Jul 30, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug... Read More

Approximately 200 people with certain rare CF mutations are now eligible for TRIKAFTA ® TORONTO , July 15, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Marketing Authorization for the expanded use of Pr TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have a mutation in... Read More

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 / Jul 02, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for... Read More

BOSTON / Jul 01, 2024 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals Second Quarter... Read More

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 <7.0% and >70% time-in-range (70-180 mg/dL), and 11 of 12 patients reduced or eliminated use of exogenous insulin 3 patients with at least 12 months of follow-up, and therefore evaluable, met the primary... Read More

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 hematopoietic stem cell transplant BOSTON / Jun 14, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced... Read More

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 study of TRIKAFTA ® showed sustained improvement in lung function at three years as well as lower rates of lung transplant and death in... Read More

BOSTON / May 22, 2024 / Business Wire / 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 Conference on Tuesday, June 4, 2024 at 4:00 p.m. CT. Charles Wagner, Executive Vice President and Chief Financial Officer, and David... Read More

BOSTON / May 15, 2024 / Business Wire / 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 as a physician, scientist and health care executive. She co-founded and serves as Chief Executive Officer of Homeward Health, a... Read More

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 submission for suzetrigine (VX-548) in moderate-to-severe acute pain and on track to complete this quarter Entered into agreement to... Read More

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 /CNW/ -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and TreeFrog Therapeutics today announced that Vertex has obtained an exclusive license to TreeFrog's... Read More

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 initiate in 2H 2024 Breakthrough Therapy designation for pain associated with DPN granted by FDA Enrollment of Phase 2 study in patients with... Read More

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 holds promise as a pipeline-in-a-product, with clinical studies in additional serious diseases underway Alpine’s protein engineering and... Read More

BOSTON / Apr 09, 2024 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals First Quarter 2024... Read More

Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review TORONTO , April 1, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission (NDS) for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent... Read More

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. BOSTON / Apr 01, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that inaxaplin (VX-147) has advanced into the Phase 3... Read More

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 company’s pipeline of potentially transformative medicines for serious diseases BOSTON / Mar 21, 2024 / Business Wire / Vertex Pharmaceuticals... Read More

BOSTON / Feb 20, 2024 / Business Wire / 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 participate in two upcoming investor conferences: TD Cowen 44 th Annual Health Care Conference on Tuesday, March 5, 2024 at 10:30 a.m.... Read More

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 / Feb 13, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY ™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY... Read More

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 submit new drug applications (NDAs) to the FDA by mid-2024 for both VX-548 in Acute Pain and the Vanzacaftor Triple in CF Broad and deep... Read More

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 potential that treating early in life may prevent disease development Vanza triple was generally well tolerated across all three studies... Read More

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 the single arm study in a broad range... Read More

BOSTON / Jan 17, 2024 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals... Read More

Approximately 1,000 patients in the U.S. 12 years of age and older are now eligible for this one-time treatment BOSTON / Jan 16, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia... Read More

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 / Jan 09, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and Drug Authority (SFDA) granted Marketing Authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy,... Read More

BOSTON / Jan 07, 2024 / Business Wire / 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 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT. “2023 was marked by continued strong performance in the cystic fibrosis business and... Read More

BOSTON / Dec 18, 2023 / Business Wire / 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. ET/8:15 a.m. PT. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section... Read More

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 eligible for treatment Approval decision from the European Commission is expected in Q1 2024 BOSTON / Dec 15, 2023 / Business Wire /... Read More

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 development in diabetic peripheral neuropathic pain following discussions with regulators VX-548 Phase 2 study in patients with painful lumbosacral... Read More

Longer-term follow-up data demonstrate consistent and durable response to treatment BOSTON / Dec 11, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from the global pivotal trials of CASGEVY™ (exagamglogene autotemcel [exa-cel]). Data from 96 patients (44 sickle cell disease [SCD], 52... Read More

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 / Dec 08, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the... Read More

BOSTON / Dec 05, 2023 / Business Wire / 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 industries, spanning drug discovery, development and commercialization. She founded and served as Chief Executive Officer of Kallyope until... Read More

TORONTO , Nov. 29, 2023 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Market Authorization for the expanded use of Pr KALYDECO ® (ivacaftor) for the treatment of cystic fibrosis (CF) in children ages 2 months and older weighing at least 3 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene: G551D ,... Read More

More than 1,200 children are newly eligible for a medicine that could treat the underlying cause of their disease BOSTON / Nov 23, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of children with cystic... Read More

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 stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available... Read More

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 continues to advance: Phase 3 data in CF and acute pain expected in early 2024; Phase 2 data in neuropathic pain expected in late 2023 BOSTON / Nov... Read More

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 / Nov 02, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that seven abstracts have been accepted for presentation at the 2023 American Society of Hematology (ASH) Annual Meeting and Exposition. This includes two... Read More

BOSTON / Nov 02, 2023 / Business Wire / 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 the 2023 North American Cystic Fibrosis Conference (NACFC) taking place November 2-4, 2023 in Phoenix, Arizona. Presentations... Read More

BOSTON / Oct 26, 2023 / Business Wire / 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 President, Global Research, and Chief Scientific Officer, will participate in a fireside chat at UBS Biopharma Conference on Thursday,... Read More

Approximately 330 children with cystic fibrosis ages 2-5 years are now eligible for a medicine that treats the underlying cause of their disease TORONTO , Oct. 17, 2023 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Market Authorization for the expanded use of Pr TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF)... Read More

BOSTON / Oct 10, 2023 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals Third Quarter 2023... Read More

BOSTON / Oct 05, 2023 / Business Wire / 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 as Chief Operating Officer and Executive Vice President of Thermo Fisher Scientific Inc. Prior to Thermo Fisher, Mr. Lagarde served... Read More

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 glucose time-in-range, and reduction or elimination of insulin use The two patients with at least 1 year of follow-up met the criteria... Read More

If approved, more than 1,200 children would be newly eligible for a medicine that could treat the underlying cause of their disease BOSTON / Sep 15, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO ®... Read More

Paul Negulescu, Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis BOSTON / Sep 14, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that three of its researchers have been awarded the 2024 Breakthrough Prize in Life Sciences “for developing life-transforming drug combinations that repair the defective... Read More

BOSTON / Aug 30, 2023 / Business Wire / 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 President and Chief Financial Officer, will participate in a fireside chat on Wednesday, September 13, 2023 at 11:30 a.m. ET. A live... Read More

BOSTON / Aug 29, 2023 / Business Wire / 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 immediately. Dr. Atkinson has served as Senior Vice President of Global Commercial Manufacturing and Supply Chain since July 2020.... Read More

First published manuscript of VX-548 preclinical and clinical trial results in a peer-reviewed journal BOSTON / Aug 03, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in the New England Journal of Medicine (NEJM) of preclinical data and the results from its Phase 2 proof-of-concept trials evaluating treatment with the selective, oral NaV1.8 inhibitor VX-548 for acute... Read More

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 granted for SCD with PDUFA date of December 8, 2023 Pipeline continues to advance with multiple near-term clinical milestones BOSTON / Aug... Read More

BOSTON / Jun 30, 2023 / Business Wire / 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. To access the call, please dial (833) 630-2124 (U.S.) or +1(412) 317-0651 (International) and reference the “Vertex Pharmaceuticals Second Quarter 2023... Read More

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 Portsmouth, New Hampshire Site BOSTON & BASEL, Switzerland / Jun 26, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and... Read More

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 severe hypoglycemic events (SHEs) and HbA1c <7.0 Both of these patients also achieved insulin independence with HbA1c values of 5.3% and... Read More

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 presentations add to the body of evidence supporting the use of CFTR modulators for all eligible people with cystic fibrosis BOSTON / Jun 09,... Read More

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, Switzerland / Jun 09, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced... Read More

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 & ZUG, Switzerland / Jun 08, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq:... Read More

BOSTON / May 30, 2023 / Business Wire / 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. Arbuckle, Executive Vice President and Chief Operating Officer, will participate in a fireside chat on Tuesday, June 13, 2023 at 2:40 p.m. PT. A live... Read More

First and only CFTR modulator approved for this age group BOSTON / May 03, 2023 / Business Wire / 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 month to less than four months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator ( CFTR... Read More

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 continues with completion of rolling BLA submissions for exa-cel in the U.S.; multiple additional clinical milestones expected in 2023 BOSTON /... Read More

About 900 children with cystic fibrosis will now have a medicine to treat the underlying cause of their disease for the first time BOSTON / Apr 26, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved the expanded use of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2... Read More

BOSTON / Apr 25, 2023 / Business Wire / 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. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the... Read More

With this approval, approximately 30 children with two copies of the F508del mutation are eligible for a medicine that treats the underlying cause of their disease for the first time TORONTO , April 10, 2023 /CNW/ - Vertex Pharmaceuticals ( Canada ) Incorporated (Nasdaq: VRTX) today announced that Health Canada, as a partner of the Access Consortium, has granted Marketing Authorization for the expanded use of Pr ORKAMBI ®... Read More

BOSTON / Apr 10, 2023 / Business Wire / 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. To access the call, please dial (877) 270-2148 (U.S.) or +1(412) 902-6510 (International) and reference the “Vertex Pharmaceuticals First Quarter 2023... Read More

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 / Apr 03, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the... Read More

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 & ZUG, Switzerland / Mar 27, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics... Read More

BOSTON / Mar 16, 2023 / Business Wire / 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 standard-of-care in people with focal segmental glomerulosclerosis (FSGS) and two APOL1 variants, a severe, rapidly progressive form of... Read More

BOSTON / Mar 09, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes (T1D). The VX-264... Read More

BOSTON / Feb 21, 2023 / Business Wire / 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 Vertex website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be... Read More

BOSTON / Feb 07, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the fourth quarter and full year ended December 31, 2022 and provided full year 2023 financial guidance. “Outstanding execution across the company resulted in another year of strong revenue growth as well as acceleration of both the research and clinical-stage pipeline,” said Reshma... Read More

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. To access the call, please dial (877) 270-2148 (U.S.) or +1(412) 902-6510 (International) and reference the “Vertex Pharmaceuticals Fourth Quarter 2022 Earnings Call”. The... Read More

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. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the... Read More

BOSTON--( BUSINESS WIRE )-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung... Read More

Entrada to receive $224 million upfront payment and $26 million equity investment, as well as potential milestone payments and royalties Global collaboration includes ENTR-701, Entrada’s EEV-investigational candidate for the treatment of DM1 BOSTON--( BUSINESS WIRE )-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Entrada Therapeutics, Inc. (Nasdaq: TRDA) today announced a global collaboration focused on discovering... Read More