Melissa Benoit

Two Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Leads to Improvements in Lung Function (FEV1) in People with Cystic Fibrosis

The research team at the Toronto Adult CF clinic would like  to share with the CF community the results of phase 3 clinical trials looking at the safety and efficacy of tezacaftor in combination with ivacaftor for patients who carry two copies of the DF508 mutation and in patients who have one copy of the DF508 mutation and a second mutation that results in residual CFTR function.  Vertex Pharmaceuticals, the sponsor of the clinical trial, announced on Tuesday, March 28, 2017 that the combination therapy results in improvement in lung function and will be moving forward with applications to health authorities like Health Canada and the FDA to allow for the sale and marketing of this drug combination.

The approval of the tezacaftor and ivacaftor combination would mean an alternative to Orkambi for patients homozygous for the DF508 mutation.

Thank you to all the patients from our centre who took part in these exciting trials. This would not be possible without their ongoing support and involvement.