The European Cystic Fibrosis Society Conference: Day 2

Director of Research Janet Allen gets an update on trials of a new drug for treating people with cystic fibrosis who have the F508del mutation, and then attends a mini symposium on ivacaftor (Kalydeco).

At the ECFS today, the most recent results for a new drug with the potential to influence the function of F508del were presented. Lumacaftor (VX809) is being developed by Vertex Pharmaceuticals to restore CFTR function to people with cystic fibrosis who have F508del. The early Phase 2 results were shown last October and based on these results, Vertex announced the launch of a Phase 3 study of lumacaftor in combination with ivacaftor (Kalydeco).

This Phase 3 trial will study two different doses of lumacaftor, 600mg once a day compared to 400mg twice a day. Both doses will be given in combination with ivacaftor (Kalydeco). A number of cystic fibrosis clinics in the UK are participating in this trial, which is recruiting people who have two copies of the F508del mutation. At the presentation today, results clearly demonstrated that both doses of lumacaftor in combination with ivacaftor had positive effects on FEV1 (increase ~8%) in the Phase 2 trial. It was also announced that the first patient had started the Phase 3 trial.

At a later session, this evening I attended a mini-symposium on ivacaftor to review the experience of physicians who have been prescribing Kalydeco for people with G551D.