Synspira Announces First Patient Dosed in Phase 1a Study of SNSP113 in Cystic Fibrosis
— First-in-class drug candidate for treatment of cystic fibrosis regardless of genetic mutation — Continue reading Phase 1a study for drug to treat CF regardless of CF Mutation
Polyphor launches the development of an inhaled antibiotic murepavadin
Polyphor Ltd has announced the development of an inhaled dosage form of its breakthrough antibiotic Murepavadin. Continue reading New Inhaled Antibiotic To Treat Respiratory Infections
Motif Bio Presents New Pre-Clinical Data for Iclaprim at IDWeek 2017™
1. Pre-Clinical Data Support the Potential Use of Iclaprim in the Treatment of Staphylococcus aureus Pneumonia in Cystic Continue reading New Pre-Clinical Data for Iclaprim
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
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.