Vertex's cystic fibrosis combination therapy Symdeko gains FDA approval

Vertex Pharmaceuticals announced that the FDA approved Symdeko, a combination of Kalydeco (ivacaftor) and tezacaftor, for the treatment of certain patients with cystic fibrosis (CF). The product, which has a list price of $292 000 per year, is indicated for treating the underlying cause of CF in people aged 12 and older who have two copies of the F508del mutation in the CFTR gene or who have at least one mutation that is responsive to the drug combination.

According to Vertex, the approval of Symdeko was based on results from the EVOLVE and EXPAND Phase III studies. In March last year, the company reported that the two trials met their primary endpoints, demonstrating significant improvements in lung function. In the EVOLVE study, there was a mean absolute improvement in percent predicted forced expiratory volume in one second through 24 weeks of 4.0 percentage points from baseline compared to placebo, while in the EXPAND trial, Symdeko demonstrated a mean absolute improvement of 6.8 percentage points versus placebo.

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Symdeko is Vertex's third medicine approved to treat the underlying cause of CF, following FDA clearance in 2015 of Orkambi (lumacaftor/ivacaftor) and the authorisation of Kalydeco in 2012. The drugmaker indicated that a marketing application for the Kalydeco and tezacaftor combination has also been submitted in Europe, with approval expected in the second half of 2018.

Vertex CEO Jeffrey Leiden remarked "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." Last month, the company announced plans to initiate late-stage studies of the experimental CF drugs VX-659 and VX-445 later this year as part of two different triple combination regimens for people with CF.

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