lumacaftor-ivacaftor for cystic fibrosis

Cystic fibrosis (CF) is a genetic disorder, caused by mutations in the cystic fibrosis transmembrane conductance regulator protein. Clinically, CF contributes to the dysfunction of multiple organs and negatively affects the airway, which makes breathing difficult. Patients with CF are also at a higher risk of infections and malnutrition. In 2015, the Food and Drug Administration and the European Medicines Agency approved the combination therapy of lumacaftor-ivacaftor for CF. Several clinical trials demonstrated that the lumacaftor-ivacaftor combination successfully improved lung function and body-mass index. Despite its success, however, some recent research suggests that lumacaftor-ivacaftor may be associated with a higher risk of discontinuation than previously appreciated.

A recent study published in the American Journal of Respiratory and Critical Care Medicine investigated the safety and effectiveness of lumacaftor-ivacaftor therapy. The authors recruited 845 CF patients, including 292 adolescents and 553 adults, from 47 centers across France. Over the course of the study, 154 patients (18.2%) had to discontinue their treatment due to adverse respiratory and non-respiratory events. This proportion is relatively high when compared to previous studies which have reported that less than 5% of patients discontinue their lumacaftor-ivacaftor treatment. Importantly, however, the individuals that continued taking the drugs were found to have an improvement in lung function and body weight. In contrast, patients that discontinued treatment were found to be at a higher risk of lung dysfunction, malnutrition, and adverse respiratory events.

The study reported that while lumacaftor-ivacaftor was associated with improved lung function and nutritional status, the proportion of patients that discontinued treatment due to adverse events was surprisingly high. The authors reasoned that the high discontinuation rate may be explained by the fact that they included a higher proportion of patients with a more severe and unstable CF disease. Additional studies with a large patient cohort are necessary to further validate the safety and effectiveness of lumacaftor-ivacaftor for CF. It is also important to note that lumacaftor-ivacaftor therapy was approved for patients with a specific CF mutation.


Written by Haisam Shah


Reference: Burgel, P. R., Munck, A., Durieu, I., Chiron, R., Mely, L., Prevotat, A., … & Marguet, C. (2019). Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. American journal of respiratory and critical care medicine, (ja).

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