Vertex’s CF drug could modify disease – long term data

Vertex has reported long-term data showing its Orkambi (lumacaftor+ivacaftor) has the potential to modify progression of cystic fibrosis in the long term.

But UK real world data of Kalydeco (ivacaftor) monotherapy in a CF subgroup failed to show significant improvement in risk of death and transplantation.

Data presented at a conference included final data from the PROGRESS 96-week extension study of the pivotal phase 3 studies of Orkambi in people aged 12 and over with two copies of the F508 del mutation.

Other highlights included real world outcomes based on patients treated with Kalydeco, drawing on data from US and UK registries.

Analysis of the 96-week PROGRESS extension study of the pivotal phase 3 TRAFFIC and TRANSPORT studies of lumacaftor+ivacaftor confirmed the safety profile, and mean lung function was maintained above baseline for up to 120 weeks.

In the US registry study data, the annual risks of death, transplantation, hospitalisation and pulmonary exacerbation were each statistically significantly lower compared to the comparator cohort of matched patients who never received ivacaftor. Trends were similar in the UK registry, but the differences in the risk of death and transplantation were not statistically significant.

However the study revealed no new safety concerns, and the majority of CF-related complications, such as CF-related diabetes and cultures positive for several microbial pathogens, were less common among ivacaftor-treated than untreated patients in both US and UK registries.

Jeffrey Chodakewitz, executive vice president and chief medical officer at Vertex, said: “The growing body of long-term data for Kalydeco and Orkambi indicates that treating the underlying cause of CF with CFTR modulators may modify the progression of this serious and life-shortening disease.”

In England, NICE has recommended National Health Service funding for Kalydeco, but not Orkambi, which was rejected in final guidance published earlier this year. Orkambi’s UK list price is £2,000 per week.


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