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Vertex makes choice for all-important CF triple therapy

Promises to treat up to 90% of CF patients


Vertex has selected what it hopes will be its biggest product yet – one which could transform cystic fibrosis (CF) treatment and the company’s fortunes.

Since launching Kalydeco in 2012, Boston, Mass-based Vertex has been breaking ground in advancing CF treatment, starting with a small subset of patients with rare mutations, but with the ultimate goal of treating all CF patients, around 75,0000 worldwide.

Its current portfolio of Kalydeco, Orkambi and Symkevi can only treat about half of this total, but up to 90% of patients would be eligible for treatment with the late-stage triple combination.

Now Vertex is on track to file its triple combination, with approval expected in the first half of 2020.

The company earned around $3bn in total revenues last year, but analysts at William Blair predict this will swell to over $10bn when the CF franchise hits its peak in the next decade, led by the forthcoming triple combination.

Vertex has had an enviable dilemma on its hands, having to choose between two similar candidates, VX-445 and VX659 to be used in combination with Symkevi (tezacaftor) and Kalydeco (ivacaftor).

Both drugs show high levels of efficacy in phase 3 trials, the key endpoint being the drug therapy’s positive impact on lung function (percent predicted forced expiratory volume in one second, ppFEV1).

Vertex announced yesterday that it had chosen VX-445. It says the decision was based on a detailed assessment of multiple factors, including favourable profiles for safety, tolerability and drug-drug interactions, the ability for co-administration with hormonal contraceptives, and the lack of photosensitivity.

The new triple combination opens up treatment for the first time to people with CF who have one F508del mutation and one minimal function mutation, and trial data shows significant improvements in these patients.

Final data announced from a 24-week phase 3 study in people with one F508del mutation and one minimal function mutation and from a 4-week phase 3 study in people with two F508del mutations will form the basis of its regulatory filings..

Data from the 24-week study in people with one F508delmutation and one minimal function mutation showed a mean absolute improvement in ppFEV1 of 14.3% points from baseline and a 63% reduction in the annualised rate of pulmonary exacerbations compared to those who received triple placebo.

In the study in people with one F508delmutation and one minimal function mutation, 2 and 0 patients, respectively, who received the VX-445 triple combination or triple placebo discontinued treatment due to adverse events. There were no discontinuations for adverse events in either arm of the study in people with two F508del mutations.

“The substantial improvements in lung function and other measures of CF seen in these phase 3 studies are unprecedented and represent a defining moment in the journey to provide medicines that treat the underlying cause of CF to the vast majority of people with CF,” said Steven M. Rowe, M.D., M.S.P.H., Director of the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham and co-chair of Vertex's triple combination steering committee.

Vertex plans to file the combination with the FDA in the third quarter of 2019, with submission to the European Medicines Agency to follow in the fourth quarter.

The company still has some significant market access obstacles in Europe, however, with ongoing rows with England and France over prices for its existing therapies, with the triple therapy also expected to hit similar affordability challenges.

Article by
Andrew McConaghie

31st May 2019

From: Marketing



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