An update on Kalydeco

Vertex pharmaceuticals, the company who pioneered the revolutionary Kalydeco drug treatment currently available for people with the GFF1D mutation – around 4% of the population, will be presenting further updates on how the drug works for people who are homozygous for the F508del mutation (the more standard mutation) at this year’s North American Cystic Fibrosis conference currently being held in Atlanta.

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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