Specialty Pharma Journal Specialty, Precision & Technology
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 …
Tag Archives: kalydeco
October, 2018
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 …
December, 2017
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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 …
August, 2017
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2 August
FDA Approves Vertex’s Kalydeco for 600+ Cystic Fibrosis Patients with One of Five Residual Mutations
BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved KALYDECO® (ivacaftor) for use in more than 600 people with cystic fibrosis (CF) ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis …
May, 2017
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18 May
FDA Expands Approval of Vertex’s Kalydeco to Treat Additional Mutations of Cystic Fibrosis
BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved KALYDECO® (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. …
March, 2017
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29 March
Vertex’s Cystic Fibrosis Combo Meets Primary Endpoints in Two Late-Stage Studies
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced results from two Phase 3 studies of the tezacaftor (VX-661) / ivacaftor combination treatment that showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in people with cystic fibrosis (CF) ages 12 and older …
December, 2016
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21 December
Galapagos Reports Positive Results From Its Phase 2 Study of Its Cystic Fibrosis Drug
Mechelen, Belgium; 20 December 2016 – Galapagos NV (Euronext & NASDAQ: GLPG) reports topline results from its SAPHIRA 1 Phase 2 study in cystic fibrosis patients with potentiator GLPG1837. The SAPHIRA 1 trial included 26 patients with the G551D mutation in CFTR each receiving three sequential doses of GLPG1837. Of …
October, 2016
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28 October
Vertex’s Orkambi and Kalydeco Show the Potential to Modify the Progression of Cystic Fibrosis
ORLANDO, Fla.–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of long-term data demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) show the potential to modify the progression of cystic fibrosis (CF). The presentations given at the 30th Annual North American Cystic Fibrosis Conference (NACFC) include final data from the …
February, 2016
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5 February
FDA Rejects Expanded Use of Vertex’s Kalydeco in Cystic Fibrosis
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO® (ivacaftor) in people with cystic fibrosis (CF) ages 2 and older who have one of …
May, 2015
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12 May
FDA Panel Recommends Approval of Vertex’s Cystic Fibrosis Combo
A US Food and Drug Administration (FDA) advisory committee has recommended approval of Vertex Pharmaceuticals’ new combination therapy for treatment of cystic fibrosis (CF). The agency’s Pulmonary-Allergy Drugs Advisory Committee voted 12 to 1 in favor of approving the drug, which consists of Vertex’s already approved drug Kalydeco (ivacaftor) and …