Articles from Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved JOURNAVX™ (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain. JOURNAVX is an effective, well-tolerated medicine without evidence of addictive potential indicated for use across all types of moderate-to-severe acute pain.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2024 financial results on Monday, February 10, 2025 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 Fourth Quarter 2024 Earnings Call.”

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 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Zai Lab Limited (Nasdaq: ZLAB; HKEX: 9688) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex’s povetacicept (pove) in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore (the licensed territory). Pove is a recombinant fusion protein therapeutic and dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) with best-in-class potential being studied for the treatment of Immunoglobulin A nephropathy (IgAN) and other B cell-mediated diseases.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 a.m. ET/7:30 a.m. PT.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis (CF) in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK. See below for Important Safety Information, including a Boxed Warning.

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 people with cystic fibrosis (CF) ages 2 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation that is responsive to TRIKAFTA based on clinical and/or in vitro data. In addition, safety information on liver injury and liver failure has been updated from warnings and precautions to a boxed warning. With this approval, 94 additional non-F508del CFTR mutations have been added to the TRIKAFTA label, and approximately 300 additional people with CF in the U.S. are now eligible for a medicine to treat the underlying cause of their disease for the first time.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced results from its Phase 2 study of suzetrigine, an investigational, oral, highly selective NaV1.8 pain signal inhibitor in people with painful lumbosacral radiculopathy (LSR). The study met its primary endpoint with statistically significant and clinically meaningful reduction in pain on the numeric pain rating scale (NPRS).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the third quarter ended September 30, 2024, and raised its full-year product revenue guidance to $10.8 billion to $10.9 billion.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided updates on multiple kidney diseases in its pipeline including IgA nephropathy (IgAN), primary membranous nephropathy (pMN), and APOL1-mediated kidney disease (AMKD). These updates demonstrate the transformative potential of Vertex’s investigational therapies in multiple serious kidney diseases, and include positive new data on povetacicept, a dual inhibitor of the BAFF and APRIL pathways, in IgAN and pMN, presented at the American Society of Nephrology’s (ASN) Kidney Week Congress on October 23-27 in San Diego, California.

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 Anesthesiologists (ASA), taking place from October 18-22, 2024 in Philadelphia, Pennsylvania.

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 Earnings Call.”

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first accepted medical presentations of the Phase 3 data on the investigational once daily vanzacaftor/tezacaftor/deutivacaftor (“vanza triple”) — the potential next-in-class triple combination medicine — will take place at the North American Cystic Fibrosis Conference (NACFC). Vertex also announced presentations describing long-term outcomes in people with cystic fibrosis (CF) ages 2 to 11 years taking TRIKAFTA®, demonstrating consistent and sustained improvements across multiple measures of disease.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the second quarter ended June 30, 2024, and raised its full year product revenue guidance to $10.65 to $10.85 billion.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) submission for suzetrigine, an investigational, oral, selective NaV1.8 pain signal inhibitor to treat moderate-to-severe acute pain. Suzetrigine has the potential to be the first new class of medicine to treat acute pain in over twenty years.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy (vanza triple) 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 conductance regulator (CFTR) gene responsive to the vanza triple. Vertex used a priority review voucher for this submission reducing the review time from 10 months to 6 months, resulting in a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025.

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 2024 Earnings Call.”

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today presented new data from its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). These updated data on 12 patients who received the full dose as a single infusion in Parts B and C of the trial are consistent with previously reported positive results in the VX-880 trial and reinforce the transformative potential of this therapy.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel [exa-cel]) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the annual European Hematology Association (EHA) Congress, confirm the transformative, consistent and durable clinical benefits of CASGEVY over time. CASGEVY is the first and only approved CRISPR-based gene-editing therapy.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also known in the European Union and in the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, were presented at this year’s European Cystic Fibrosis Society’s (ECFS) 47th European Cystic Fibrosis Conference held June 5-8, 2024, in Glasgow, Scotland.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences.

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 company committed to rearchitecting the delivery of health and care for nearly 60 million Americans living in rural America. Prior to founding Homeward in 2022, Dr. Schneider served as President and Chief Medical Officer of Livongo Health, a biotechnology company serving people with chronic conditions including diabetes, hypertension, obesity and behavioral health issues. While there, she led the company through the largest consumer digital health Initial Public Offering in history and the industry’s largest merger between Livongo and Teladoc Health. She also served as Chief Medical Officer of Castlight Health, a health care navigation company for self-insured companies.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the first quarter ended March 31, 2024, and reiterated full year 2024 financial guidance.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced important advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades. Suzetrigine is an oral selective NaV1.8 pain signal inhibitor (formerly known as VX-548).

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 Earnings Call.”

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that inaxaplin (VX-147) has advanced into the Phase 3 portion of the global Phase 2/3 pivotal clinical trial in APOL1-mediated kidney disease (AMKD), where a 45 mg once daily oral dose will be compared to placebo, on top of standard of care. The clinical trial is designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with proteinuric kidney disease mediated by two variants in the APOL1 gene, known as AMKD. In addition, the trial has been expanded to include adolescents with AMKD ages 10 to 17 years.

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-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants. ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease.

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:

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 is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the fourth quarter and full year ended December 31, 2023 and provided full year 2024 financial guidance.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program, the most comprehensive Phase 3 pivotal program ever conducted by Vertex for the treatment of cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein. The Phase 3 program included two randomized, double-blind, active-controlled, 52-week trials, SKYLINE 102 and SKYLINE 103, evaluating the efficacy of vanzacaftor (20 mg)/tezacaftor (100 mg)/deutivacaftor (250 mg) once daily in people with CF ages 12 years and older who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators (CFTRm), compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor). A third Phase 3 single-arm, 24‑week, open-label study, RIDGELINE 105, evaluated the safety and efficacy of the vanza triple in children with CF ages 6 to 11 years with at least one mutation responsive to triple combination CFTRm.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its Phase 3 program for the selective NaV1.8 inhibitor, VX-548, in the treatment of moderate-to-severe acute pain. The Phase 3 program included two randomized, double-blind, placebo-controlled, pivotal trials, one following abdominoplasty surgery and one following bunionectomy surgery, as well as a single arm safety and effectiveness study which enrolled patients with a broad range of surgical and non-surgical pain conditions.

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 Fourth Quarter 2023 Earnings Call.”

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 (TDT) in patients 12 years and older.

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, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). CASGEVY is approved for the treatment of people 12 years of age and older with SCD or TDT. The Kingdom of Saudi Arabia has among the highest prevalence rates of SCD and TDT in the world, with thousands of patients living with these genetic blood disorders.

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 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its Phase 2 dose-ranging study of the selective NaV1.8 inhibitor VX-548 in people with painful diabetic peripheral neuropathy (DPN). Treatment with all doses of VX-548 resulted in a statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the weekly average of daily pain intensity on a Numeric Pain Rating Scale (NPRS) at Week 12. The study also included an active reference arm of pregabalin to support the evaluation of the VX-548 treatment effect.

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]).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs.

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 2021 and remains on its Board of Directors and is Chair of Research & Development. Prior to joining Kallyope, Ms. Thornberry spent more than 30 years at Merck & Co., Inc., most recently as Senior Vice President and Global Franchise Head, Diabetes and Endocrinology, with responsibility for discovery and clinical research in diabetes, obesity and women’s health. Among her many notable scientific accomplishments, Ms. Thornberry initiated and led Merck’s dipeptidyl peptidase 4 (DPP-4) project, which resulted in the discovery and commercialization of JANUVIA® for the treatment of type 2 diabetes, the best-selling oral product franchise in Merck's history. She also identified the first caspase, interleukin-1β converting enzyme (ICE/caspase-1).

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 fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the third quarter ended September 30, 2023, raised full year 2023 guidance for product revenue and reiterated full year 2023 guidance for operating expenses.

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 presentations of updated clinical data describing additional patients with longer follow-up duration from pivotal Phase 3 trials investigating exagamglogene autotemcel’s (exa-cel) potential as a one-time functional cure for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

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 will highlight results from two open-label extension studies demonstrating the long-term clinical benefits and safety profile of TRIKAFTA® among CF pediatric populations.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences.

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 Earnings Call.”

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 as President and Chief Operating Officer of Patheon, a contract manufacturer to the biopharma industry, and as a Managing Director at JLL Partners, a private equity firm investing in health care services.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today presented longer-term data on patients dosed in Parts A and B of its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). Prior to VX-880 treatment, all six patients enrolled had long-standing T1D with no endogenous insulin secretion, required an average of 34.0 units of insulin per day, and had a history of recurrent severe hypoglycemic events (SHEs) in the year prior to screening.

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® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

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 chloride channel protein in patients with cystic fibrosis.” Specifically, the award celebrates the work of Paul Negulescu, Ph.D., Fredrick Van Goor, Ph.D., and Sabine Hadida, Ph.D., who have worked together to lead cystic fibrosis (CF) discovery for over 20 years.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Morgan Stanley 21st 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.

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. This new role and function unifies responsibility for small molecule, cell and gene therapies across preclinical, clinical and commercial biopharmaceutical sciences, manufacturing operations and global supply chain.

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 pain following abdominoplasty and bunionectomy surgeries.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the second quarter ended June 30, 2023 and updated its full year 2023 financial guidance.

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 Earnings Call.”

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Lonza (SIX: LONN) today announced a strategic collaboration to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with T1D, currently focusing on the VX-880 and VX-264 programs that are currently in clinical trials.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today presented data on all patients dosed in Parts A and B of its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). All six patients treated with VX-880 had undetectable fasting C-peptide (endogenous insulin secretion) at baseline, a history of recurrent SHEs in the year prior to treatment and required an average of 34.0 units of insulin per day. Following treatment, all six patients demonstrated endogenous insulin secretion, improved glycemic control as measured by HbA1c, improved time-in-range on continuous glucose monitoring, and reduction or elimination of exogenous insulin use. Patients with greater than 90 days of follow-up had elimination of SHEs in the evaluation period.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 12 scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines were presented at this year’s European Cystic Fibrosis Society's (ECFS) 46th European Cystic Fibrosis Conference held June 7-10, 2023, in Vienna, Austria. Together, the data presented show the long-term benefits of treatment with CFTR modulators as well as the importance of treating the underlying cause of CF as early in life as possible. Key data presented at this year’s conference are highlighted below.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that both pivotal trials for exagamglogene autotemcel (exa-cel) in patients with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) met primary and key secondary endpoints at pre-specified interim analyses. The results are being presented at the Annual European Hematology Association (EHA) Congress.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Goldman Sachs 44th 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.

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) gene that is responsive to KALYDECO® based on clinical and/or in vitro assay data. KALYDECO® is already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the first quarter ended March 31, 2023 and reiterated full year 2023 financial guidance.

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 through 5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to TRIKAFTA® based on in vitro data. TRIKAFTA® was previously approved by the FDA for use in people with CF 6 years and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive to TRIKAFTA® based on in vitro data.

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.

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 Earnings Call.”

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 investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The BLAs include requests for Priority Review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D).

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 chronic kidney disease also known as APOL1-mediated kidney disease (AMKD). AMKD is caused by variants of the APOL1 gene and affects approximately 100,000 people in the U.S. and Europe. Inaxaplin is an APOL1 inhibitor aimed at treating the underlying cause of AMKD.