The FDA approved a third disease-modifying cystic fibrosis drug product from Vertex Pharmaceuticals to treat the underlying cause of the disease, the company announced Monday.
To be sold as Symdeko, it’s a two-tablet product combining ivacaftor (Kalydeco) with a new agent called tezacaftor. One tablet, to be taken in the morning, contains both drugs; the second contains ivacaftor only and is to be taken in the evening. The product was approved for patients 12 and older who have two copies of the F508del mutation in the CFTR gene or some other mutation that responds to tezacaftor/ivacaftor.
In a press release, the company announced that it would begin shipping Symdeko to pharmacies across the United States this week.
Results from two company-sponsored phase II studies showed the newly approved, combination treatment to be associated with statistically significant and and clinically meaningful improvements in lung function and other measures of disease, with a favorable safety profile in CF patients with two copies of the F508del mutation or one F508del mutation and one mutation resulting in residual CFTR function.
The EVOLVE and the EXPAND study findings were published last November in the New England Journal of Medicine.