The FDA has approved tezacaftor/ivacaftor and ivacaftor (Symdeko, Vertex Pharmaceuticals, Inc.) to treat the underlying cause of cystic fibrosis (CF) in patients 12 years of age and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. Symdeko is Vertex’s third medicine approved to treat the underlying cause of CF. The company reports that it will begin shipping it to pharmacies in the United States this week.
According to a BioPharma Dive report, Vertex has set Symdeko’s annual list price at $292,000, which analysts predict may net the company about $100 million to $200 million from the new drug this year.
In November 2017, the New England Journal of Medicine published the results of two phase 3 studies of Symdeko. These studies—EVOLVE and EXPAND—enrolled approximately 750 people 12 years of age and older with CF 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 favorable safety profile. The most common adverse events, regardless of treatment group, included infective pulmonary exacerbation and cough.
The first data from the ongoing EXTEND rollover study, also presented in November, show that the lung function improvements and the safety and tolerability profiles seen in EVOLVE and EXPAND were sustained for up to 48 total weeks of Symdeko treatment.
The European Medicines Agency has validated the marketing authorization application for the tezacaftor/ivacaftor combination, and Vertex expects approval in the European Union in the second half of 2018.