With antibiotic-resistant “superbugs” continuing to be a threat in U.S. hospitals, doctors are looking for innovative ways to cut down on disease transmission. Continue reading Docs May Have Trick Up Their Sleeves Fighting Germs
Savara Announces Start of Pivotal Phase III AVAIL Study of AeroVanc
Savara, Inc.(NASDAQ: SVRA), a clinical-stage specialty pharmaceutical company focused on the development Continue reading Phase III study starts AVAIL study
Jeffrey Leiden, chief executive for Vertex Pharmaceuticals, plans to eradicate cystic fibrosis. (Vertex) Continue reading Vertex Plans To Eradicate Cystic Fibrosis, And Go From There
A year removed from its initial grant of $525,000, Cystic Fibrosis Foundation Therapeutics (CFFT) is providing $3 million in additional funding to 4D Molecular Therapeutics (4DMT). Continue reading 4D Molecular Receives $3 Million from Cystic Fibrosis Foundation Therapeutics
ProQR Completes Dosing of Cystic Fibrosis Patients in QR-010 Phase 1b Trial
• Last patient received their final dose in the PQ-010-001 Phase 1b clinical trial of QR-010 in CF patients with the F508del mutation.
Top-line trial data are expected to be issued in a press release Continue reading Phase 1b Trial of QR-010
Published: Aug. 29, 2017
Michael Maiden, a Michigan State University College of Osteopathic Medicine student and doctoral candidate in the Department of Microbiology and Molecular Genetics, was Continue reading Fighting an old enemy in the battle against cystic fibrosis
Els van der Heijden, who has cystic fibrosis, was finding it ever harder to breathe as her lungs filled with thick, sticky mucus. Despite taking more than a dozen pills and inhalers a day, the 53-year-old had to stop Continue reading Lab-made “mini organs” helping doctors treat cystic fibrosis
Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis
The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.
The NDA submission was based on positive results from 2 global Phase 3 trials, which showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in patients treated with tezacaftor/ivacaftor.
The combination treatment consists of ivacaftor (marketed under the brand name Kalydeco), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and tezacaftor, a novel CFTR corrector. Tezacaftor is designed to address the processing defect of F508del-CFTR to enable it to reach the cell surface, where ivacaftor can further enhance the protein’s function.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of February 28, 2018 to make a decision on the NDA.
For more information visit Vrtx.com.
ProQR Announces Results for the Second Quarter of 2017
• Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic Continue reading Two clinical trials presented at the European Cystic Fibrosis Conference