Vertex's latest cystic fibrosis combination approved in US

Vertex’s quest to treat a wider group of patients with cystic fibrosis (CF) continues after the FDA approved its latest combination therapy, Symdeko.

Symdeko adds the new component of tezacaftor to the previously approved drug ivacaftor, making this the company’s third product for the life-threatening lung disorder.

Vertex wants to use the combination as a backbone for eventual triple combinations that could treat up to 90% of patients with the life-shortening disease that leads to serious lung infections and deteriorating respiratory function.

For now, Symdeko and previously approved Kalydeko and Orkambi cover around 45% of the population.

The company recently released data from two proposed triple combinations showing a statistically significant improvement in lung function, as measured by a standard air volume exhalation test.

But the treatment will not come cheap – the list price for Symdeko will be $22,400 for a 28-day supply, bringing the annual treatment cost to $292,000.

Symdeko is approved in patients aged 12 or over who have two copies of the F508del mutation in the CFTR gene, who have at least one mutation that is responsive to the drug.

Approval was based on the phase 3 EVOLVE and EXPAND studies. This involved around 750 people with CF aged 12 and older with two copies of the F508del mutation, or with one F508del mutation and one mutation that results in residual CFTR function.

Across both studies, patients treated with Symdeko experienced statistically significant and clinically meaningful improvements in lung function and other measures of disease, with a favourable safety profile.

The most common adverse events, regardless of treatment group, included infective pulmonary exacerbation and cough.

Vertex is also studying Symdeko in the EXTEND rollover study – first data presented in November showed that lung function improvements and safety seen in EVOLVE and EXPAND were sustained for to 48 weeks of treatment.

European regulators are also reviewing Symdeko and are expected to make a decision the second half of this year.

Vertex last month selected two ‘next-generation correctors’, VX-659 and VX-445 to move into phase 3 trials.

These will form a three-drug combination that would mean the company can provide therapies covering 90% of patients with CF.

Vertex's Jeffrey Leiden

Vertex’s CEO, Jeffrey Leiden, said: “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."