Specialty Pharma Journal Specialty, Precision & Technology
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 …
Tag Archives: cystic fibrosis
March, 2017
-
27 March
Genetics Reveal Mysteries of Hard-to-Treat Bacterial Infection in Cystic Fibrosis
New UBC research on bacteria that cause major problems for those with cystic fibrosis reveals clues as to how it proliferates for so long in the lungs and offers new ideas for treatments to explore. “Someone with cystic fibrosis has about one less teaspoon of water in the mucous in …
-
6 March
Vertex Acquires Cystic Fibrosis Candidate from Concert Pharmaceuticals in Potential $250 Million Deal
BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has signed a definitive asset purchase agreement to acquire CTP-656 from Concert Pharmaceuticals (Nasdaq: CNCE). CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination …
-
3 March
PTC Therapeutics Discontinues Ataluren Program After Failed Phase 3 Trial in Nonsense Mutation Cystic Fibrosis
SOUTH PLAINFIELD, N.J., March 2, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, …
January, 2017
-
23 January
Concert Pharmaceuticals’ Cystic Fibrosis Candidate Receives Orphan Drug Status from the FDA
LEXINGTON, Mass.–(BUSINESS WIRE)– Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for CTP-656, Concert’s next generation CFTR potentiator being developed for the treatment of cystic fibrosis. In December 2016, Concert initiated a Phase 2 trial in the U.S. …
-
4 January
RaNA Therapeutics Acquires Shire’s Messenger RNA Platform
CAMBRIDGE, Mass.–(BUSINESS WIRE)–RaNA Therapeutics, a leader in the discovery and development of a new class of medicines that target RNA, today announced the acquisition of the MRT platform, an mRNA therapy platform, developed by subsidiaries of Shire plc. In addition, Shire’s former MRT employees, the group focused on the development …
December, 2016
-
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
-
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 …
September, 2016
-
12 September
Horizon to Acquire Rare Disease Company Raptor Pharmaceutical for $800 Million
DUBLIN, Ireland and NOVATO, Calif., Sept. 12, 2016 (GLOBE NEWSWIRE) — Horizon Pharma plc (NASDAQ:HZNP) and Raptor Pharmaceutical Corp. (NASDAQ:RPTP) today announced the companies have entered into a definitive agreement under which Horizon Pharma will acquire all of the issued and outstanding shares of Raptor Pharmaceutical Corp. common stock for …
August, 2016
-
17 August
Vertex to Stop of Experimental Cystic Fibrosis Phase 3 Combo Trial
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided an update on its ongoing Phase 3 development program of its investigational compound VX-661 in combination with ivacaftor, which includes four studies that together are expected to enroll more than 1,000 people with cystic fibrosis (CF). Based on a planned interim futility …