Vertex submits Kalydeco combo for cystic fibrosis

Vertex has submitted a new combination treatment for the rare genetic disease cystic fibrosis (CF) to the US FDA and the European Medicines Agency (EMA).

The therapy combines Vertex's already approved CF treatment Kalydeco (ivacaftor) with a new drug lumacaftor, which is known as a CFTR corrector.

Vertex is seeking approval for the use of the combination in a specific subset of CF patients who have two copies of a genetic mutation known as F508del. According to Vertex, there are 8,500 people with CF who are aged 12 and older who have two copies of this mutation in the US and around 12,000 in Europe.

Kalydeco broke new ground in CF when it was launched in 2012 as it was the first drug to tackle the underlying genetic cause of the disease rather than its symptoms, which include the development of a thick mucus that affects a number of organs, particularly the lungs and digestive system.

The drug has led Vertex's revenues since its launch due to the lack of alternative CF treatments, recording revenues of $127m during the third quarter of 2014. Vertex's total revenues for the period were $179m.

Approval for the combination therapy would encourage this growth to continue, while CF patients would benefit from another treatment option in area of high unmet medical need.

Due in part to this unmet need and the severity of CF – the average survival age is around 37 – the combination of Kalydeco and lumacaftor received a breakthrough designation from the FDA in 2012.

Vertex is seeking additional review advantages from the FDA, including a priority review designation, which, if granted, would shorten the assessment time from one year to eight months.

The combination treatment has already been granted accelerated assessment in Europe, shortening the EMA's review time from around 210 days to 150 days.

Kalydeco was the first drug approved to treat the underlying cause of cystic fibrosis

Both submissions are based on phase III data from the TRAFFIC and TRANSPORT studies, as well as interim data from a subsequent rollover study involving people who have two copies of the F508del mutation.

According to Vertex, people treated with the combination therapy showed improvements in lung function and other measures of disease, such as pulmonary exacerbation, through 24 weeks of treatment.

The rollover study has since shown lung function improvements were sustained for 48 total weeks of treatment.

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