New Cystic Fibrosis Research Data From Vertex Reveals Further Value of Kalydeco

Vertex Pharmaceuticals will be presenting 15 abstracts at the 28th Annual North American Cystic Fibrosis Conference (NACFC) held in Atlanta from October 9-11. Each abstract is from Vertex’s cystic fibrosis research and development program. Data will primarily be related to TRAFFIC and TRANSPORT clinical trials, which were phase 3 rollover studies evaluating lumacaftor and ivacaftor combination therapy.

According to a news release from Vertex, presented data will include the first interim data from patients who completed TRAFFIC and TRANSPORT, which were highlighted previously on Cystic Fibrosis News Today. These studies were for individuals homozygous for the F508del mutation. Data from this subtype of cystic fibrosis patients, who were on combination lumacaftor and ivacaftor therapy for 24 weeks, were previously presented and will be revisited at NACFC. Data concerning combination therapy will be related to drug distribution in the body, safety in children on the treatment, and the effects of using bronchodilators while on concomitant combination therapy.

Presented studies will also focus on children receiving ivacaftor treatment. These studies evaluated individuals with R117H residual functional mutations who benefit from the potenitation mechanism of ivacaftor. Topics include the effects of ivacaftor on forced expiratory volume in one second (FEV1), the distribution of ivacaftor throughout the body following administration, and the effects of ivacaftor on patient weight.

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Also of note is a study concerning “sodium bromide pulse as a biomarker of multi-orgran CFTR function in human subjects.” Although this was not a study explicitly concerning ivacaftor and lumacaftor, the results have relevance in monitoring cystic fibrosis.

According to the release, all abstracts are available in the online edition of Pediatric Pulmonology. Invited speakers and symposium session are also listed, as well as a list of posters.