Indacaterol was an effective once-daily bronchodilator and was at least as effective as tiotropium in improving clinical outcomes for patients with COPD. Clinical trial registered with clinicaltrials.gov (NCT 00463567).

Background A light-porous-particle, dry-powder formulation of tobramycin was developed, using PulmoSphere® technology, to improve airway delivery efficiency, substantially reduce delivery time, and improve patient convenience and satisfaction. We evaluated the safety, efficacy and convenience of tobramycin inhalation powder (TIP™) versus tobramycin inhalation solution (TIS, TOBI®) for treating Pseudomonas aeruginosa infection in cystic fibrosis (CF) patients aged ≥6 years. Methods In this open-label study, 553 patients were randomized 3:2 to TIP (total 112 mg tobramycin) via the Novartis T-326 Inhaler or TIS 300 mg/5 mL via PARI LC® PLUS nebulizer twice daily for three treatment cycles (28 days on-drug, 28 days off-drug). Safety, efficacy, and treatment satisfaction outcomes were evaluated. Results TIP was generally well-tolerated; adverse events were similar in both groups. The rate of cough suspected to be study-drug related was higher in TIP-treated patients (TIP: 25.3%; TIS: 4.3%), as was the overall discontinuation rate (TIP: 26.9%; TIS: 18.2%). Increases in FEV1 % predicted from baseline to Day 28 of Cycle 3 were similar between groups; mean reduction in sputum Pseudomonas aeruginosa density (log10 CFU/g) on Day 28 of Cycle 3 was also comparable between groups. Administration time was significantly less for TIP (mean: 5.6 versus 19.7 minutes, p<0.0001). Treatment satisfaction was significantly higher for TIP for effectiveness, convenience, and global satisfaction. Conclusions TIP has a safety and efficacy profile comparable with TIS, and offers a far more convenient treatment option for pseudomonas lung infection in CF.

Ivacaftor was efficacious in a group of patients with CF who had selected non-G551D gating mutations.

Background Indacaterol is a long-acting inhaled b 2 -

Background Ivacaftor, approved for treatment of cystic fibrosis (CF) patients aged ≥6 years with G551D-CFTR or other gating mutations, was evaluated in subjects with R117H-CFTR, a residual function mutation. Methods A 24 week, placebo-controlled, double-blind, randomized clinical trial (RCT) enrolled 69 CF subjects aged ≥6 years with R117H-CFTR and percent predicted forced expiratory volume in 1 second (ppFEV1) ≥40. Primary outcome was absolute change from baseline in ppFEV1 through week 24. Secondary outcomes included sweat chloride, CF Questionnaire-Revised (CFQ-R) respiratory domain, and safety. An open-label extension enrolled 65 RCT subjects after washout; after 12 weeks, an interim analysis was performed. Findings After 24 weeks, treatment difference in mean absolute change in ppFEV1 between ivacaftor (n=34) and placebo (n=35) was 2.1 percentage points (p=0·20). Ivacaftor treatment resulted in significant RCT treatment differences in sweat chloride (−24.0 mmol/L; p<0.001) and CFQ-R respiratory domain (8.4; p=0.009). In prespecified subgroup analyses, ppFEV1 significantly improved with ivacaftor in subjects aged ≥18 years (treatment difference vs placebo: 5.0 percentage points; p=0.01), but not in subjects aged 6 to 11 years (−6.3 percentage points; p=0.03). In the extension study, both placebo/ivacaftor and ivacaftor/ivacaftor groups showed ppFEV1 improvement (absolute change from postwashout baseline at week 12: 5.5 percentage points; p<0.0001). No new safety concerns were identified. Interpretation Although this RCT did not meet the primary outcome, secondary outcomes and subgroup analyses suggest that ivacaftor significantly improves lung function in adult patients with R117H-CFTR and may benefit patients with established disease.