Ataluren is a cystic fibrosis transmembrane conductance regulator stimulant currently under development for the treatment of cystic fibrosis with a nonsense mutation. It is only suitable for a small number of patients with this specific gene mutation.
Other results reported for this study (only abstract available) were:
• The number of pulmonary exacerbations did not differ significantly between treatment groups (rate ratio 0.77 [95% CI 0.57-1.05]; p=0.0992).
• However, post-hoc analysis of the subgroup of patients not using chronic inhaled tobramycin showed a 5.7% difference (95% CI 1.5-10.1) in relative change from baseline in percent-predicted FEV1 between the ataluren and placebo groups at week 48 (-0.7% [-4.0 to 2.1] vs −6.4% [-9.8 to -3.7]; nominal p=0.0082), and fewer pulmonary exacerbations in the ataluern group (1.42 events [0.9-1.9] vs 2.18 events [1.6-2.7]; rate ratio 0.60 [0.42-0.86]; nominal p=0.0061).
Based on this particular result, the authors suggest ataluren might be beneficial for patients not taking chronic inhaled tobramycin.
Safety profiles were generally similar for ataluren and placebo, except for the occurrence of increased creatinine concentrations (acute kidney injury), which occurred in 18 (15%) out of 118 patients in the ataluren group vs. 1 (<1%) out of 120 patients in the placebo group. No life-threatening adverse events or deaths were reported in either group.