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Combination therapy: Kalydeco+VX809
- Question
- Following recent results presented at the NACFC in Orlando, it seems that the association Kalydeco+VX809 is promising. Will the drug be commercially available in 2015/2016?
- Answer
- At the recent NACFC meeting, investigators presented the results from a preliminary (Phase II) study assessing the effect and tolerance of VX809 at different doses alone or in combination with Ivacaftor (Kalydeco) in patients with two deltaF 508 mutations. Results from this short-term (2 months) study performed in a limited number (n=82) of patients showed that although VX809 had only limited effects on lung function (FEV1) and sweat chloride concentrations, combination of high doses of VX809 + Ivacftor resulted in a small but significant increase in FEV1 and in reduction of sweat chloride concentrations. These promising results will require confirmation in a larger study over a longer period of time (Phase III). Until the results of this study are available, it is impossible to conclude that this approach will be successful and to provide a date range for marketing of this drug.
Dr Pierre-Régis Burgel - 12.11.2012
