VERTEX PHARMACEUTICALS INCORPORATED


Associated tags: Biotechnology, Health, Pharmaceutical, Vertex Pharmaceuticals, Pharmaceutical industry, Vertex, Genetics, Clinical Trials, VRTX, Science, Research, Patient

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, February 13, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Hospital, European Commission, CRISPR, Volatile organic compound, Hematology, Gene, HSC, VRTX, TDT, SCD, European, Civil service commission, Haematopoietic system, Patient, Sickle cell disease, Bambino, Catholic higher education, Pediatrics, Vertex Pharmaceuticals, Pharmaceutical industry

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.

Key Points: 
  • 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 the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment.
  • Through this work, Vertex has secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY.

Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis

Retrieved on: 
Monday, February 5, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CFTR, Patient, Consultant, Safety, VRTX, Mutation, Vertex, Chairperson, Disease, Therapy, Cystic fibrosis, Medicine, University, Vertex Pharmaceuticals

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.

Key Points: 
  • 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.
  • In both SKYLINE 102 and SKYLINE 103, the primary endpoint of absolute change from baseline in ppFEV1 through week 24 was met and showed that treatment with vanza triple was non-inferior to treatment with TRIKAFTA.
  • The results from other secondary endpoints were consistent with results of the primary and key secondary endpoints.
  • Treatment with the vanza triple was well tolerated in all three studies, and the safety was similar between the vanza triple and TRIKAFTA treatment groups in SKYLINE 102 and SKYLINE 103.

Vertex Announces Positive Results From the VX-548 Phase 3 Program for the Treatment of Moderate-to-Severe Acute Pain

Retrieved on: 
Tuesday, January 30, 2024
Health, FDA, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Multimedia, Abdominoplasty, Pain, Patient, VRTX, Vertex Pharmaceuticals

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.

Key Points: 
  • 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.
  • For the first key secondary endpoint, Vertex tested the hypothesis that VX-548 was superior to hydrocodone bitartrate/acetaminophen (HB/APAP) on SPID48 following abdominoplasty surgery or bunionectomy surgery.
  • VX-548 had a more rapid onset to meaningful pain relief than placebo in both the abdominoplasty and bunionectomy trials.
  • “We are very pleased with the results from the VX-548 pivotal program, which demonstrate a compelling and consistent combination of efficacy and safety across multiple acute pain conditions and settings.

Vertex to Announce Fourth Quarter and Full Year 2023 Financial Results on February 5

Retrieved on: 
Wednesday, January 17, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Vertex Pharmaceuticals, Webcast, Vertex, International, VRTX

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.

Key Points: 
  • 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.
  • 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.”
    The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section.
  • To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast.

Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024
Health, FDA, Genetics, General Health, Clinical Trials, Pharmaceutical, Biotechnology, SCD, Vertex Pharmaceuticals, Patient, Hospital, CRISPR, Haematopoietic system, Food, Sickle cell disease, TDT, PDUFA, VRTX, Pharmaceutical industry

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.

Key Points: 
  • 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.
  • “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com .

Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia

Retrieved on: 
Tuesday, January 9, 2024
Health, Genetics, Clinical Trials, General Health, Pharmaceutical, Biotechnology, SCD, Vertex Pharmaceuticals, Patient, KFSH, Kingdom, Hospital, CRISPR, Authorization, Medicine, Sickle cell disease, TDT, SFDA, Marketing, ATC, VRTX, Prevalence, Pharmaceutical industry

CASGEVY is approved for the treatment of people 12 years of age and older with SCD or TDT.

Key Points: 
  • 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 is working to qualify additional hospitals as ATCs to bring CASGEVY to patients, including the King Faisal Specialist Hospital (KFSH).
  • In order to enable rapid access to CASGEVY, Vertex is working to secure listing on hospital formularies to support reimbursement as soon as possible.

Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings

Retrieved on: 
Sunday, January 7, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, SCD, Kingdom, Blue Shield, Conference, T1D, Sickle cell disease, Gene editing, Blue Cross, Pain, Messenger, EMA, FDA, PDUFA, Cystic fibrosis, Insulin, Vertex Pharmaceuticals, Abdominoplasty, European Medicines Agency, Sciatica, SAD, PNP, Glycated hemoglobin, Patient, TDT, Safety, Diabetes, Acceleration, Hypoglycemia, CFTR, VRTX, CHMP, Pharmaceutical industry

Epidemiology update: Vertex revised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada.

Key Points: 
  • Epidemiology update: Vertex revised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada.
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of March 30, 2024, for CASGEVY in TDT.
  • VX-993: Vertex also anticipates initiating a Phase 2 study with an oral formulation of VX-993 for the treatment of PNP in 2024.
  • 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.

Vertex to Present at the 42nd Annual J.P. Morgan Healthcare Conference on January 8

Retrieved on: 
Monday, December 18, 2023
Health, Stem Cells, Genetics, Research, Science, Pharmaceutical, Biotechnology, News, Conference, VRTX, Vertex Pharmaceuticals

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.

Key Points: 
  • 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.
  • 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 company's website.

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Friday, December 15, 2023
Research, FDA, Genetics, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Quality, Pediatrics, European Commission, Sickle cell disease, European Medicines Agency, HSC, Bambino, Vertex, Civil service commission, Patient, European, Pediatric Hematology and Oncology, CHMP, Gene, Committee, Advanced Therapy Medicinal Product, Hematology, VRTX, CRISPR, TDT, Beta thalassemia, Volatile organic compound, Catholic higher education, SCD, Quality of life, Vertex Pharmaceuticals, Pharmaceutical industry

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).

Key Points: 
  • 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).
  • An approval decision by the European Commission is expected in February 2024.
  • “This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex.
  • “As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”