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: CF
November, 2018
October, 2018
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25 October
AbbVie Assumes Full Development and Commercial Control of Galapagos’ Cystic Fibrosis Assets
NORTH CHICAGO, Ill., Oct. 24, 2018 /PRNewswire/ — AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, announced today that it will assume full development and commercial responsibility for its collaboration with Galapagos (Euronext: GLPG) to discover and develop new therapies to treat cystic fibrosis (CF). AbbVie’s research program aims to develop a …
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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 Grants Orphan Drug Designation to Synspira’s Investigational Cystic Fibrosis Therapy
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Synspira, a privately held company developing a new class of inhaled glycopolymer-based therapeutics for the treatment of pulmonary disease, today announced that it has been granted Orphan Designation by the United States Food and Drug Administration (FDA) for poly (acetyl, arginyl) glucosamine (PAAG15A), for the treatment of cystic …
August, 2018
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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 …
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3 August
Celtaxsys’ Cystic Fibrosis Drug Demonstrated Clinically Meaningful Improvements in Pulmonary Exacerbations in Phase 2 Trial
ATLANTA, Aug. 02, 2018 (GLOBE NEWSWIRE) — Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare inflammatory diseases, today announced top line results of its Phase 2 EMPIRE-CF trial evaluating oral, once daily anti-inflammatory molecule, acebilustat, for the treatment of cystic fibrosis (CF), …
June, 2018
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29 June
Galapagos Announces Topline Results from Cystic Fibrosis Study and Says AbbVie will not Proceed with Second CF Combo
Mechelen, Belgium; 28 June 2018; 22.35 CET; regulated information – Galapagos NV (Euronext & NASDAQ: GLPG) announces the topline results with investigational C2 corrector GLPG2737 in the first Phase 2 CF patient trial with this candidate and provides an update on its triple combo development strategy. The PELICAN study was designed …
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 …
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12 April
FDA to Allow Translate Bio to Proceed with Phase 1/2 Clinical Trial in Patients with Cystic Fibrosis
LEXINGTON, Mass.–(BUSINESS WIRE)–Translate Bio, a leading messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company to begin a first-in-human clinical trial of MRT5005 …