Vertex gets approval for combo-therapy in cystic fibrosis


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All eyes are on Vertex’s two Phase 3 trials into a triple therapy for cystic fibrosis (CF), in the meantime, however, it’s just received the nod from the FDA for its two therapy combination treatment in the same area.

Symdeko, as the treatment is called, combines existing treatment Kalydeco (ivacafto) with tezacaftor. The combination treatment was shown to improve lung function, reduced pulmonary exacerbations, increase BMI (though in a non-statistically significant manner) and patients reported fewer symptoms, such as coughing and wheezing, in a Phase 3 trial.

The treatment will not come cheap, with a list price of $292,000 per year – with sales of the new therapy expected to range between $100 million to $500 million through to 2020.

“Today is an exciting day for the CF community. The approval of Symdeko, our third disease-modifying CF medicine, offers many patients an important new treatment option,” said Jeffrey Leiden, Vertex’s Chairman, President and Chief Executive Officer. “This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting.”

Symdeko is added to Vertex’s two other treatments, Orkambi and standalone Kalydeco. It will be used in patients who are aged 12 and over, who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or who have at least one mutation that is responsive to tezacaftor/ivacafto.

Vertex released that it expects the treatment to also receive approval by the EMA in the second half of 2018.

Beyond this, the major point of excitement for the biotech will be on the read out from its two Phase 3 trials into its triple-therapy in CF. It already scored positive data in Phase 2 for tezacaftor and Kalydeco, alongside VX-659, which posted an improvement to lung function by 13.3%.

It will also test the two drugs alongside another candidate, VX-445, giving the biotech two options for potential success in trials.

Ben Hargreaves