Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis, as Reported By the Study’s Author

Oct 14, 2019 | Clinical Trials, Cystic Fibrosis, France, Real-Life Study

Pierre-Régis Burgel MD, PhD, Professor of Respiratory Medicine at Paris Descartes University and Coordinator of French National Reference Center for Cystic Fibrosis at Cochin Hospital, interviewed with regarding the study of lumacaftor-ivacaftor improving the lung and nutritional status of patients with cystic fibrosis. The study was funded by the French CF Patient Association “Vaincre la Mucoviscidose”, the French CF learning Society, and Legs Pascal Bonnet. It was conducted independently from the lumacaftor-ivacaftor manufacturer. Below are the takeaways.

When asked about the background of this study and how lumacaftor-ivacaftor differs from other treatments for CF, Dr. Burgel said, “Safety and efficacy of lumacaftor-ivacaftor, which partially restores CFTR function, have been reported in phase 3 clinical trials in patients 12 years of age or older who had CF and were homozygous for the Phe508del. Improvement in lung function, reduction in pulmonary exacerbations and a trend towards an increase in body mass index (BMI) led to its approval by the Food and Drug Administration in February 2015 and by the European Medicines Agency in November 2015. However, the magnitude of effect on percent predicted forced expiratory volume in 1 sec (ppFEV1), the small improvement in nutritional status and the limited use of concomitant treatment for reducing exacerbations have cast doubt on the clinical benefits associated with lumacaftor-ivacaftor, which has not been approved in several countries.”

The study is being conducted at 47 pediatric and/or adult centers to evaluate the effects of lumacaftor-ivacaftor treatment in a real-life setting in France.

The main findings, according to Dr. Burgel, “were driven by patients who received prolonged (continuous or intermittent) exposure to lumacaftor-ivacaftor, whereas patients in whom lumacaftor-ivacaftor was discontinued had a significant decrease in ppFEV1, no increase in body weight or BMI and no decrease in the use of IV antibiotics. Importantly, treatment discontinuation was less prevalent in adolescents than in adults and the results suggest that the magnitude of lung function improvement could be greater in adolescents.”

Readers should keep in mind that “these data further indicate that the benefits and risks of new therapies cannot be extrapolated to patients who are excluded from clinical trials. Further, findings from the present study concur with the concept that starting CFTR modulators earlier in life could be an important strategy.”


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