Specialty Pharma Journal Specialty, Precision & Technology
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that treatment with the triple combination of the next-generation corrector VX-659, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). …
Tag Archives: vertex
November, 2018
October, 2018
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19 October
Vertex Data Presented at NACFC Demonstrate Rapid Progress Toward Expanding and Enhancing Options for Treating Cystic Fibrosis
DENVER–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that eight scientific abstracts from the company’s portfolio of cystic fibrosis (CF) medicines are being presented at the 32nd North American Cystic Fibrosis Conference taking place October 18-20, 2018 in Denver. Key highlights include presentations of Phase 2 data evaluating clinical safety and efficacy …
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11 October
FDA Lifts Clinical Hold on Vertex/CRISPR Therapeutics’ Investigational Gene Therapy CTX001 for the Treatment of Sickle Cell Disease
ZUG, Switzerland and BOSTON and CAMBRIDGE, Mass., Oct. 10, 2018 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of …
August, 2018
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30 August
Vertex and Genomics plc Collaborate to Use Human Genetics and Data Science to Advance Discovery of Precision Medicines
BOSTON & OXFORD, United Kingdom–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Genomics plc today announced a three-year collaboration, extendable to five years, to use human genetics and machine learning to improve discovery of targets for precision medicines, and to advance understanding of the clinical impact of human genetic variation and …
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16 August
FDA Approves Kalydeco for Treatment of Cystic Fibrosis in Children Ages 12 to <24 Months with Certain Mutations in the CFTR Gene
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO …
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9 August
FDA Approves Vertex’s Orkambi to Treat Cystic Fibrosis Patients Ages 2-5 Years with Most Common Form of Disease
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF …
June, 2018
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1 June
FDA Puts Clinical Hold on CRISPR and Vertex’s IND Application for CTX001 for the Treatment of Sickle Cell Disease
BOSTON and ZUG, Switzerland and CAMBRIDGE, Mass., May 30, 2018 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be …
April, 2018
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27 April
Vertex Initiates Late-Stage Studies for its Triple Combination Regimen for Patients with Cystic Fibrosis
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it is initiating two Phase 3 studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF). The first Phase 3 study will evaluate approximately 360 people with CF who have one copy of the F508del mutation …
December, 2017
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13 December
Vertex and CRISPR Therapeutics Partner to Develop CTX001 as CRISPR/Cas9 Gene Edited Treatment
BOSTON, CAMBRIDGE, Mass. & ZUG, Switzerland–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR Therapeutics AG (NASDAQ: CRSP) today announced that the companies will co-develop and co-commercialize CTX001, an investigational gene editing treatment, as part of the companies’ previously announced collaboration aimed at the discovery and development of new gene editing treatments …
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7 December
Vertex’s Kalydeco Demonstrates Positive Late-Stage Results in Children with Cystic Fibrosis Ages 1 to 2 Years
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from an open-label Phase 3 study of KALYDECO® (ivacaftor) in children with cystic fibrosis (CF) ages 1 to 2 years who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The ARRIVAL study met its primary endpoint …